A new Phase 2 study at Children’s National will look at using the drug vosoritide to promote growth in children with growth disorders.
A child’s growth is often measured by pediatricians during routine physicals to identify abnormalities of growth and stature. An abnormality in these measurements could mean a child has a growth disorder. There are many different causes of growth disorders. Some can be the result of defects in genes related to the growth plate, which is the tissue near the end of long bones that grows as the child grows. Children with a growth disorder can present many different symptoms including short stature, joint pain, heart problems, bone problems and developmental delays. Scientists still have a lot to learn about what exactly causes these genetic growth disorders and treatments are limited, especially in the pediatric population. Growth hormone is not uniformly helpful and has only been approved for a small number of conditions.
Vosoritide is an investigational drug that directly targets the growth plate to promote growth. It is an analog of the amino acid C-type natriuretic peptide (CNP). It binds its receptor on healthy cartilage cells called chondrocytes and is currently under investigation in clinical trials as a treatment for the bone growth disorder achondroplasia.
“Vosoritide for Selected Genetic Causes of Short Stature” is a Phase 2 study currently open at Children’s National Hospital. This study will target five types of genetic short stature including SHOX deficiency, hypochondroplasia, Rasopathies (which includes Noonan syndrome), heterozygous NPR2 defects and CNP deficiency.
Thirty-five children with short stature will be enrolled and followed for a six-month observation period to obtain baseline growth velocity, safety profile and quality of life assessment. Study participants will then be treated with vosoritide for 12 months and will be assessed for safety and improvement in growth outcomes.
“Many patients who present with short stature likely have genetic defects in genes involved in growth plate physiology. Those patients with selected causes of short stature that interact with the CNP pathway may benefit from treatment with vosoritide, which directly targets the growth plate,” said Andrew Dauber, M.D., MMSc., lead investigator of this clinical study and chief of Endocrinology at Children’s National Hospital, a program ranked in the top 10 by U.S. News & World Report. “In this study, our goal is to understand if vosoritide may be a safe and effective treatment option for certain genetically defined short stature syndromes.”
This clinical trial has been approved by the FDA and funded by BioMarin. Children’s National is the only site in the world offering this therapy for patients with these conditions. The study is currently underway and subject recruitment is ongoing. There are 9 participants enrolled to date.
“This study could fundamentally change the way we treat certain growth disorders”, says Dr. Dauber.
For more information on the clinical trial, click here.
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Children’s National Hospital announced a $5 million gift from The Boeing Company that will help drive lifesaving pediatric discoveries at the new Children’s National Research & Innovation Campus.
Children’s National Hospital announced a $5 million gift from The Boeing Company that will help drive lifesaving pediatric discoveries at the new Children’s National Research & Innovation Campus. The campus, now under construction, is being developed on nearly 12 acres of the former Walter Reed Army Medical Center. Children’s National will name the main auditorium in recognition of Boeing’s generosity.
“We are deeply grateful to Boeing for their support and commitment to improving the health and well-being of children in our community and around the globe,” said Kurt Newman, M.D., president and CEO of Children’s National “The Boeing Auditorium will help the Children’s National Research & Innovation campus become the destination for discussion about how to best address the next big healthcare challenges facing children and families.”
The one-of-a-kind pediatric hub will bring together public and private partners for unprecedented collaborations. It will accelerate the translation of breakthroughs into new treatments and technologies to benefit kids everywhere.
“Children’s National Hospital’s enduring mission of positively impacting the lives of our youngest community members is especially important today,” said Boeing President and CEO David Calhoun. “We’re honored to join other national and community partners to advance this work through the establishment of their Research & Innovation Campus.”
Children’s National Research & Innovation Campus partners currently include Johnson & Johnson Innovation – JLABS, Virginia Tech, the National Institutes of Health (NIH), Food & Drug Administration (FDA), U.S. Biomedical Advanced Research and Development Authority (BARDA), Cerner, Amazon Web Services, Microsoft, National Organization of Rare Diseases (NORD) and local government.
The 3,200 square-foot Boeing Auditorium will be the focal point of the state-of-the-art conference center on campus. Nationally renowned experts will convene with scientists, medical leaders and diplomats from around the world to foster collaborations that spur progress and disseminate findings.
Boeing’s $5 million commitment deepens its longstanding partnership with Children’s National. The company has donated nearly $2 million to support pediatric care and research at Children’s National through Chance for Life and the hospital’s annual Children’s Ball. During the coronavirus pandemic, Boeing fabricated and donated 2,000 face shields to help keep patients and frontline care providers at Children’s National safe.
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The Children’s National Hospital Turner Syndrome Clinic is proud to be recognized by the Turner Syndrome Global Alliance (TSGA) as a Level 4 Regional Resource Center. Level 4 is the highest Level of Care designation and is based on the KidNECT Care Model which encourages family networking, education, comprehensive coordinated care and transition support as well as leadership in Turner Syndrome (TS) research.
TS is a rare genetic disorder that occurs in 1 to about 2,500 girls and is caused by a partial or complete missing X chromosome. Some of the characteristics of TS are short stature, delayed puberty, kidney, thyroid and heart problems. Although there is no cure for TS, many of the symptoms can be treated.
The Children’s National TS Clinic is part of the Division of Endocrinology and Diabetes which is ranked by U.S. News & World Report as one of the top 10 programs in the nation. The TS Clinic opened in January 2019 and is the first one-of-its-kind in the Washington, D.C. region. A multidisciplinary clinic is held once a month with the team comprising of cardiology, endocrinology, psychology, gynecology and genetics to help care for the needs of patients with TS all in one day. The referral network of specialties includes neuropsychology, otolaryngology, audiology, orthopedics, urology and dentistry.
“I am so proud of our team for their hard work and the excellent clinical care they provide for girls with Turner Syndrome,” says Roopa Kanakatti Shankar, M.D., endocrinologist at Children’s National. “This recognition by the Turner Syndrome Global Alliance means that we not only provide comprehensive care but also serve as a regional leader and resource center for the families we serve. We will continue to raise awareness about Turner Syndrome through our research and partnerships.”
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Randi Streisand, Ph.D., is the recipient of the 2020 Richard R. Rubin Award. This national award, sponsored by the American Diabetes Association, recognizes a scientist who has made outstanding, innovative contributions to the study and understanding of the behavioral aspects of diabetes.
Currently, Dr. Streisand is a tenured professor of psychology and pediatrics at The George Washington University School of Medicine and serves as chief of the Division of Psychology & Behavioral Health at Children’s National Hospital. Additionally, she serves as vice-chair for the Institutional Review Board, and provides clinical services for families impacted by diabetes and other health conditions. She is also an active mentor and educator for trainees and early career faculty members regarding research and clinical service.
Throughout her career, Dr. Streisand has received multiple research and training grants, published approximately 100 papers in peer-reviewed journals, and presented at national and international meetings. She has also been recognized for her mentorship and received awards from Children’s National as well as the Society of Pediatric Psychology. Dr. Streisand’s research is funded by the National Institutes of Health (NIH) and focuses on parent-child adjustment to diabetes, and behavioral interventions to promote adjustment, adherence and glycemic control. Her research has included young children through teenagers, and she is currently evaluating the use of a parent coach program to support parents of young children newly diagnosed with type 1 diabetes.
Dr. Streisand has served on several editorial boards for journals, serves as a grant reviewer for NIH, and has served in elected positions for professional organizations. She is also a Certified Diabetes Educator.
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Growth hormone therapy is one of the most common treatments for short stature in children. However, endocrinologists report mixed outcomes, even when children have the same underlying condition as the cause of their short stature. Despite research into a variety of potential causes for this unpredictable response, there is still very little scientific evidence to help physicians predict whether children with short stature who are treated with growth hormone will respond to the treatment or not.
“Previous disease-specific models have been developed using multiple clinical variables such as parental height, age at treatment start, peak hormone levels and doses, and birth parameters, however, these clinical parameters only partially predict variation in response,” wrote the study authors, including Andrew Dauber, M.D., first author and chief of the division of Endocrinology at Children’s National Hospital. “Our goal was to perform a large-scale genome-wide study to provide a comprehensive assessment of how common genetic variation may play a role in growth hormone response.”
To accomplish this, the study combined five cohorts from across the world to identify 614 individuals for further review. All patients had short stature caused by conditions including growth hormone deficiency, small for gestational age, or idiopathic short stature (no previously identified cause), who received growth hormone as treatment.
Interestingly, the researchers found no overwhelming genetic predictors of response to growth hormone. They did identify a few signals that may potentially play a role in the body’s response to growth hormone but noted those signals will need further exploration. The study also ruled out the idea that genetic predictors of height in the general population might predict response to growth hormone.
“Identifying genetic predictors of how a child with short stature will respond to growth hormone would be an important step forward for clinicians and parents to make informed decisions about the right treatment approach,” says Dr. Dauber. “Though we didn’t find any specific indicators, this large-scale study has allowed us to rule out some previously held assumptions and offers several new avenues to explore.”
The study was conducted in collaboration with Pfizer and Boston Children’s Hospital.
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Children’s National Hospital in Washington, D.C., was ranked No. 7 nationally in the U.S. News & World Report 2020-21 Best Children’s Hospitals annual rankings. This marks the fourth straight year Children’s National has made the list, which ranks the top 10 children’s hospitals nationwide.
In addition, its neonatology program, which provides newborn intensive care, ranked No.1 among all children’s hospitals for the fourth year in a row.
For the tenth straight year, Children’s National also ranked in all 10 specialty services, with seven specialties ranked in the top 10.
“Our number one goal is to provide the best care possible to children. Being recognized by U.S. News as one of the best hospitals reflects the strength that comes from putting children and their families first, and we are truly honored,” says Kurt Newman, M.D., president and CEO of Children’s National Hospital.
“This year, the news is especially meaningful, because our teams — like those at hospitals across the country — faced enormous challenges and worked heroically through a global pandemic to deliver excellent care.”
“Even in the midst of a pandemic, children have healthcare needs ranging from routine vaccinations to life-saving surgery and chemotherapy,” said Ben Harder, managing editor and chief of Health Analysis at U.S. News. “The Best Children’s Hospitals rankings are designed to help parents find quality medical care for a sick child and inform families’ conversations with pediatricians.”
The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals. The rankings recognize the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News. The top 10 scorers are awarded a distinction called the Honor Roll.
The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.
Below are links to the seven Children’s National specialty services that U.S. News ranked in the top 10 nationally:
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Children’s National Research Institute directors Vittorio Gallo, Ph.D., and Mark Batshaw, M.D.
The Children’s National Research Institute recently released its 2019-2020 academic annual report, titled 150 Years Stronger Through Discovery and Care to mark the hospital’s 150th birthday. Not only does the annual report give an overview of the institute’s research and education efforts, but it also gives a peek in to how the institute has mobilized to address the coronavirus pandemic.
“Our inaugural research program in 1947 began with a budget of less than $10,000 for the study of polio — a pressing health problem for Washington’s children at the time and a pandemic that many of us remember from our own childhoods,” says Vittorio Gallo, Ph.D., chief research officer at Children’s National Hospital and scientific director at Children’s National Research Institute. “Today, our research portfolio has grown to more than $75 million, and our 314 research faculty and their staff are dedicated to finding answers to many of the health challenges in childhood.”
Highlights from the Children’s National Research Institute annual report
In 2018, Children’s National began construction of its new Research & Innovation Campus (CNRIC) on 12 acres of land transferred by the U.S. Army as part of the decommissioning of the former Walter Reed Army Medical Center campus. In 2020, construction on the CNRIC will be complete, and in 2012, the Children’s National Research Institute will begin to transition to the campus.
In late 2019, a team of scientists led by Eric Vilain, M.D., Ph.D., director of the Center for Genetic Medicine Research, traveled to the Democratic Republic of Congo to collect samples from 60 individuals that will form the basis of a new reference genome data set. The researchers hope their project will generate better reference genome data for diverse populations, starting with those of Central African descent.
A gift of $5.7 million received by the Center for Translational Research’s director, Lisa Guay-Woodford, M.D., will reinforce close collaboration between research and clinical care to improve the care and treatment of children with polycystic kidney disease and other inherited renal disorders.
The Center for Neuroscience Research’s integration into the infrastructure of Children’s National Hospital has created a unique set of opportunities for scientists and clinicians to work together on pressing problems in children’s health.
Children’s National and the National Institute of Allergy and Infectious Diseases are tackling pediatric research across three main areas of mutual interest: primary immune deficiencies, food allergies and post-Lyme disease syndrome. Their shared goal is to conduct clinical and translational research that improves what we know about those conditions and how we care for children who have them.
An immunotherapy trial has allowed a little boy to be a kid again. In the two years since he received cellular immunotherapy, Matthew has shown no signs of a returning tumor — the longest span of time he’s been tumor-free since age 3.
In the past 6 years, the 104 device projects that came through the National Capital Consortium for Pediatric Device Innovation accelerator program raised $148,680,256 in follow-on funding.
Even though he’s watched more than 500 aspiring physicians pass through the Children’s National pediatric residency program, program director Dewesh Agrawal, M.D., still gets teary at every graduation.
Understanding and treating the novel coronavirus (COVID-19)
In a short period of time, Children’s National Research Institute has mobilized its scientists to address COVID-19, focusing on understanding the virus and advancing solutions to ameliorate the impact today and for future generations. Children’s National Research Institute Director Mark Batshaw, M.D., highlighted some of these efforts in the annual report:
Eric Vilain, M.D., Ph.D., director of the Center for Genetic Medicine Research, is looking at whether or not the microbiome of bacteria in the human nasal tract acts as a defensive shield against COVID-19.
Catherine Bollard, M.D., MBChB, director of the Center for Cancer and Immunology Research, and her team are seeing if they can “train” T cells to attack the invading coronavirus.
Sarah Mulkey, M.D., Ph.D., an investigator in the Center for Neuroscience Research and the Fetal Medicine Institute, is studying the effects of, and possible interventions for, coronavirus on the developing brain.
You can view the entire Children’s National Research Institute academic annual report online.
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A diabetes clinic gives undocumented children with diabetes access to the care they need to maintain their health with this chronic condition.
In 2018, Jennifer Boughton, a social worker at Children’s National Hospital, came up with the idea of starting a diabetes clinic for patients whose immigration status prevented them from qualifying for insurance. The idea came about after undocumented children were arriving in the emergency department with high blood sugar and dangerously elevated ketone levels.
Through donations and the employees, who volunteer their time, Boughton’s idea became a reality. In January of 2019, the diabetes center held its first access clinic. The clinic has been open every three months since then for undocumented children with diabetes.
Flyers for the clinic are sent to local schools and organizations to help recruit patients.
In the first year, the patient volume has quadrupled and the hbA1c levels have decreased for patients who attend.
Unfortunately, we’ve been notified that the ENDO2020 conference has been canceled due to concerns of COVID-19. Because of this, we will not be hosting our reception in honor of Andrew Dauber, M.D., on Sunday, March 29.
We hope to see you at a future Endocrinology or Pediatric Endocrinology event.
Children’s National Hospital is incredibly proud of the work Dr. Dauber has done in the endocrinology community.
Andrew Dauber, M.D., MMSc, division chief of Endocrinology at Children’s National Hospital, will be awarded the 2020 Richard E. Weitzman Outstanding Early Career Investigator Award at ENDO 2020. The prestigious award will be presented at the annual meeting of the Endocrine Society in recognition of Dauber’s work in understanding the regulation of growth and puberty, and applying innovative genetic technologies to studying pediatric endocrinology. Dauber credits many collaborators throughout the world, as well as the team at Children’s National for the award.
With a five-year grant from the National Institutes of Health (NIH), Dauber and colleagues from the Cincinnati Children’s Hospital Medical Center, Boston Children’s Hospital and the Children’s Hospital of Philadelphia are using electronic health records to identify children who likely have rare genetic growth disorders. Using cutting-edge DNA sequencing technologies, including whole exome sequencing, the researchers are aiming to identify novel genetic causes of severe growth disorders. The first paper describing genetic findings in patients with high IGF-1 levels was published in Hormone Research in Paediatrics in December 2019.
Dauber and researchers at Cincinnati Children’s Hospital Medical Center are exploring how to treat patients with mutations in the PAPPA2 gene. In 2016, the group described the first patients with mutations in this gene who had decreased the bioavailability of IGF-1, stunting their growth and development. In their current phase of research, findings are emphasizing the importance of this gene in regulating IGF-1 bioavailability throughout childhood. The ultimate aim is to create therapies to increase IGF-1 bioavailability, thereby supporting healthy growth and development in children. Their first study to track PAPPA2 and intact IBGBP-3 concentrations throughout childhood was published in the European Journal of Endocrinology in January 2020.
Dauber is particularly interested in studying children with dominantly inherited forms of short stature. Along with collaborators in Cincinnati, he currently has an ongoing treatment trial using growth hormone in patients with Aggrecan gene mutations. Dauber hopes to announce soon a new clinical trial for children with all forms of dominantly inherited short stature.
Study upon study has shown us that there are many factors that affect an individual’s height and growth. As these studies and the conversation around how to identify and address genomic anomalies become more prevalent, the team at Children’s National is increasingly interested in engaging with other centers around the country. In the coming months, the Children’s National Research & Innovation Campus will open on the grounds of the former Walter Reed Army Medical Center, which will serve as a one-of-a-kind pediatric research and innovation hub. A critical component to this campus is the co-location of Children’s National research with key partners and incubator space.
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This past fall, Nadia Merchant, M.D., joined Children’s National Hospital as an endocrinologist in the Endocrinology and Diabetes Department. Dr. Merchant received her undergraduate and medical education at Weill Cornell Medical College in Qatar. She completed her pediatric residency at Wright State Boonshoft School of Medicine. She then completed her genetics residency and pediatric endocrine fellowship at Baylor College of Medicine/Texas Children’s Hospital.
Dr. Merchant was born with acromesomelic dysplasia, a rare genetic disorder, but that hasn’t stopped her from pursuing her medical career. While at Baylor College of Medicine, Dr. Merchant was very active in quality improvement projects, research and organizations that raise awareness of endocrine related conditions. For several years, she was a moderator at Baylor College of Medicine for “From Stress to Strength,” at a course for parents of children with genetic disorders and autism. Dr. Merchant also served as an endocrine fellow representative on the American Academy of Pediatrics Section on Endocrinology (SOEn) for the last two years and also served on the committee for a Bone and Mineral special interest group within the Pediatric Endocrine Society (PES). During medical school, she worked with Positive Exposure, an organization that uses visual arts to celebrate human diversity for individuals living with genetic, physical, behavioral and intellectual differences.
Dr. Nadia Merchant is currently one of the endocrinologists in the multidisciplinary bone health clinic at Children’s National, a clinic dedicated to addressing and improving bone health in children. Dr. Merchant also manages endocrine manifestations in children with rare genetic disorders.
The Endocrinology department at Children’s National is ranked among the best in the nation by “U.S. News & World Report”.
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Fewer than half of California pharmacies provided correct prescription drug disposal details, a percentage that dropped if “secret shoppers” made their call on a weekend, according to a brief research report published online Dec. 31, 2019, in Annals of Internal Medicine.
The callers pretended to be well-meaning parents who were trying to safely dispose of unneeded antibiotics and opioid-based prescription painkillers after their child’s surgery. Fewer than half of the California pharmacies they called provided correct prescription drug disposal details, a percentage that dropped sharply if the “secret shoppers” made their call on a weekend, according to a brief research report published online Dec. 31, 2019, in Annals of Internal Medicine.
“The Food and Drug Administration advises consumers about how to safely dispose of unneeded medicines and, because pharmacists can play an integral role in this conversation, the American Pharmacists Association says prescription medication disposal should follow FDA guidelines,” says Rachel E. Selekman, M.D., MAS, a pediatric urologist at Children’s National Hospital and the study’s first author. “We found very few California pharmacies permitted take-back of unneeded medications. There was also a striking difference in the accuracy and completeness of drug disposal information depending on whether they answered the call on a weekday or a weekend. That suggests room for improvement,” Dr. Selekman says.
The multi-institutional research team, led by Primary Investigator and senior author Hillary L. Copp, M.D., MS, at University of California, San Francisco, identified licensed pharmacies located in urban and rural settings in California. That state that accounts for 10% of all U.S. pharmacies. They wrote a script that guided four male and two female “secret shoppers” to ask about what to do about leftover antibiotics (sulfamethoxazole-trimethoprim tablets) and a liquid opioid-based painkiller (hydrocodone-acetaminophen). From late-February to late-April 2018, they called 898 pharmacies from 8 a.m. to 8 p.m., asking about the correct way to dispose of these medicines.
According to the FDA, consumers should mix most unused medicines with an unappealing substance, like kitty litter, place it in a sealed container and toss the container in the trash. Medicines that can be harmful to others, like opioids, should be flushed down the sink or toilet. Many pharmacies have programs or kiosks to handle unused prescription medicines.
Of the pharmacies surveyed in California:
47% provided correct information about disposing of antibiotics
29% provided correct information about how to dispose of both antibiotics and opioids
19% provided correct information about how to dispose of opioids
49% provided correct antibiotic disposal information and 20% provided correct opioid disposal information on weekday calls
15% provided correct antibiotic disposal information and 7% provided correct opioid disposal information on weekend calls
Asked specifically about drug take-back programs, just 11% said their pharmacy had one that could be used to dispose of antibiotics or opioids.
“Unused prescription medications can be misused by others and can result in accidental childhood poisonings,” Dr. Selekman adds. “The bottom line is that we often talk about how to address the problem of too many unused medications lingering in homes. There are many reasons this is a problem, but part of the problem is nobody knows what to do if they have too many prescription medicines. Because of this research, we have discovered that pharmacies don’t uniformly provide accurate information to our patients. Patients, families and health care professionals who advise families should work together to help improve and expand safe disposal options for these powerful medications.”
In addition to Drs. Selekman and Copp, the research team includes co-authors Thomas W. Gaither, M.D., MAS, Zachary Kornberg, BA, and Aron Liaw, M.D., all of whom were at the University of California, San Francisco, School of Medicine, Division of Pediatric Urology at the time the study was performed.
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Brynn Marks, M.D., endocrinologist at Children’s National Hospital, was diagnosed with Type 1 diabetes at the age of five years old and knows too well the struggles that may come with managing this chronic condition. After finding the right, knowledgeable provider as a teen, Dr. Marks realized that she wanted to become an endocrinologist and be that resource and support for others with Type 1.
Developments in diabetes technologies, including continuous glucose monitors (CGM) and insulin pumps, hold great promise for improving diabetes control while also improving quality of life for those living with Type 1. However, the pace of development also presents challenges for busy clinicians who must keep up with rapid developments in the field. Dr. Marks is focused on making sure patients and providers have the latest information and training on diabetes technologies with the goal that they can be more effectively used by more people in the real world.
“These diabetes technologies are very important for kids and teens with diabetes because they can help them to live life as normally as possible while affording the freedom they need to just be kids,” says Dr. Marks.
Dr. Marks’ recent research includes a study where she used an app to deliver medical education about diabetes technology. Participants received authentic, case-based scenarios focused on the technologies to help them apply knowledge of these technologies to real-world clinical scenarios involving insulin pumps and CGMs. All of the education was delivered through questions and explanations in an effort to keep the learners engaged with the curriculum. The questions were repeated over 3-4 months to improve learning and long-term knowledge retention. The study showed that knowledge and confidence about these technologies improved and ultimately led to better patient care.
Dr. Marks presented two posters at a recent International Society for Pediatric and Adolescent Diabetes (ISPAD) meeting related to her efforts to make these technologies more accessible:
Experiential Learning in T1D Technology Education: Knowledge of Parents and Clinicians: Dr. Marks enrolled different groups of learners in the diabetes technology curriculum mentioned above, including parents of children with Type 1 diabetes, attending physicians, pediatric endocrine fellows and certified diabetes educators. Results showed minimal difference among those groups in terms of knowledge about the pumps and CGMs. Interestingly, clinicians who had the opportunity to wear the technologies for educational purposes had greater knowledge than clinicians who did not have the same opportunity. Based on these results, Dr. Marks advocated that opportunities to wear pumps and CGMs should be a routine part of education for clinicians working with these technologies.
A Qualitative Analysis of Clinicians’ Experiences Wearing CGM: Dr. Marks explored the reactions of clinicians without diabetes who were given the opportunity to wear continuous glucose monitors for 1 week. Participants reported that the opportunity to wear these technologies improved their knowledge and gave them greater empathy for patients using CGM.
Dr. Marks’ work to date has identified strategies to improve knowledge about insulin pumps and CGM. Moving forward, she will continue to study the best ways to educate parents and clinicians about these diabetes technologies in hopes of improving the day to day lives of the children they care for.
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The brain comprises only 2% of the body’s volume, but it uses more than 20% of its energy, which makes this organ particularly vulnerable to changes in metabolism.
More than 30 million Americans have diabetes, with the vast majority having Type 2 disease. Characterized by insulin resistance and persistently high blood sugar levels, poorly controlled Type 2 diabetes has a host of well-recognized complications: compared with the general population, a greatly increased risk of kidney disease, vision loss, heart attacks and strokes and lower limb amputations.
But more recently, says Nathan A. Smith, MS, Ph.D., a principal investigator in Children’s National Research Institute’s Center for Neuroscience Research, another consequence has become increasingly apparent. With increasing insulin resistance comes cognitive damage, a factor that contributes significantly to dementia diagnoses as patients age.
The brain comprises only 2% of the body’s volume, but it uses more than 20% of its energy, Smith explains – which makes this organ particularly vulnerable to changes in metabolism. Type 2 diabetes and even prediabetic changes in glucose metabolism inflict damage upon this organ in mechanisms with dangerous synergy, he adds. Insulin resistance itself stresses brain cells, slowly depriving them of fuel. As blood sugar rises, it also increases inflammation and blocks nitric oxide, which together narrow the brain’s blood vessels while also increasing blood viscosity.
When the brain’s neurons slowly starve, they become increasingly inefficient at doing their job, eventually succumbing to this deprivation. These hits don’t just affect individual cells, Smith adds. They also affect connectivity that spans across the brain, neural networks that are a major focus of his research.
While it’s well established that Type 2 diabetes significantly boosts the risk of cognitive decline, Smith says, it’s been unclear whether this process might be halted or even reversed. It’s this question that forms the basis of a collaborative Frontiers grant, $2.5 million from the National Science Foundation split between his laboratory; the lead institution, Stony Brook University; and Massachusetts General Hospital/Harvard Medical School.
Smith and colleagues at the three institutions are testing whether changing the brain’s fuel source from glucose to ketones – byproducts from fat metabolism – could potentially save neurons and neural networks over time. Ketones already have shown promise for decades in treating some types of epilepsy, a disease that sometimes stems from an imbalance in neuronal excitation and inhibition. When some patients start on a ketogenic diet – an extreme version of a popular fat-based diet – many can significantly decrease or even stop their seizures, bringing their misfiring brain cells back to health.
Principal Investigator Smith and his laboratory at the Children’s National Research Institute are using experimental models to test whether ketones could protect the brain against the ravages of insulin resistance. They’re looking specifically at interneurons, the inhibitory cells of the brain and the most energy demanding. The team is using a technique known as patch clamping to determine how either insulin resistance or insulin resistance in the presence of ketones affect these cells’ ability to fire.
They’re also looking at how calcium ions migrate in and out of the cells’ membranes, a necessary prerequisite for neurons’ electrical activity. Finally, they’re evaluating whether these potential changes to the cells’ electrophysiological properties in turn change how different parts of the brain communicate with each other, potentially restructuring the networks that are vital to every action this organ performs.
Colleagues at Athinoula A. Martinos Center for Biomedical Imaging at Massachusetts General Hospital and Harvard Medical School, led by Principal Investigator Eva-Maria Ratai, Ph.D., will perform parallel work in human subjects. They will use imaging to determine how these two fuel types, glucose or ketones, affect how the brain uses energy and produces the communication molecules known as neurotransmitters. They’re also investigating how these factors might affect the stability of neural networks using techniques that investigate the performance of these networks both while study subjects are at rest and performing a task.
Finally, colleagues at the Laufer Center for Physical and Quantitative Biology at Stony Brook University, led by Principal Investigator Lilianne R. Mujica-Parodi, Ph.D., will use results generated at the other two institutions to construct computational models that can accurately predict how the brain will behave under metabolic stress: how it copes when deprived of fuel and whether it might be able to retain healthy function when its cells receive ketones instead of glucose.
Collectively, Smith says, these results could help retain brain function even under glucose restraints. (For this, the research team owes a special thanks to Mujica-Parodi, who assembled the group to answer this important question, thus underscoring the importance of team science, he adds.)
“By supplying an alternate fuel source, we may eventually be able to preserve the brain even in the face of insulin resistance,” Smith says.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2019/12/brain-network-illustration.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2019-12-12 10:58:442022-05-09 15:49:21$2.5M to protect the brain from metabolic insult
Recently, Kurt Newman, M.D., president and CEO of Children’s National Hospital, authored an opinion piece for the popular political website, The Hill. In the article, he called upon stakeholders from across the landscape to address the significant innovation gap in children’s healthcare versus adults.
As Chair of the Board of Trustees of the Children’s Hospital Association, Dr. Newman knows the importance of raising awareness among policy makers at the federal and state level about the healthcare needs of children. Dr. Newman believes that children’s health should be a national priority that is addressed comprehensively. With years of experience as a pediatric surgeon, he is concerned by the major inequities in the advancements of children’s medical devices and technologies versus those for adults. That’s why Children’s National is working to create collaborations, influence policies and facilitate changes that will accelerate the pace of pediatric healthcare innovation for the benefit of children everywhere. One way that the hospital is tackling this challenge is by developing the Children’s National Research & Innovation Campus, which will be the nation’s first innovation campus focused on pediatric research.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2018/02/Kurt-Newman-capitol-building.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2019-12-11 15:41:002023-07-10 10:07:22Making healthcare innovation for children a priority
Children’s National Hospital and Virginia Tech create formal partnership that includes the launch of a Virginia Tech biomedical research facility within the new Children’s National Research & Innovation Campus.
Children’s National Hospital and Virginia Tech recently announced a formal partnership that will include the launch of a 12,000-square-foot Virginia Tech biomedical research facility within the new Children’s National Research & Innovation Campus. The campus is an expansion of Children’s National that is located on a nearly 12-acre portion of the former Walter Reed Army Medical Center in Washington, D.C. and is set to open its first phase in December 2020. This new collaboration brings together Virginia Tech, a top tier academic research institution, with Children’s National, a U.S. News and World Report top 10 children’s hospital, on what will be the nation’s first innovation campus focused on pediatric research.
“Virginia Tech is an ideal partner to help us deliver on what we promised for the Children’s National Research & Innovation Campus – an ecosystem that enables us to accelerate the translation of potential breakthrough discoveries into new treatments and technologies,” says Kurt Newman, M.D., president and CEO, Children’s National. “Our clinical expertise combined with Virginia Tech’s leadership in engineering and technology, and its growing emphasis on biomedical research, will be a significant advance in developing much needed treatment and cures to save children’s lives.”
Earlier this year, Children’s National announced a collaboration with Johnson & Johnson Innovation LLC to launch JLABS @ Washington, DC at the Research & Innovation Campus. The JLABS @ Washington, DC site will be open to pharmaceutical, medical device, consumer and health technology companies that are aiming to advance the development of new drugs, medical devices, precision diagnostics and health technologies, including applications in pediatrics.
“We are proud to welcome Virginia Tech to our historic Walter Reed campus – a campus that is shaping up to host some of the top minds, talent and innovation incubators in the world,” says Washington, D.C. Mayor Muriel Bowser. “The new Children’s National Research & Innovation Campus will exemplify why D.C. is the capital of inclusive innovation – because we are a city committed to building the public and private partnerships necessary to drive discoveries, create jobs, promote economic growth and keep D.C. at the forefront of innovation and change.”
Faculty from the Children’s National Research Institute and the Fralin Biomedical Research Institute at Virginia Tech Carilion (VTC) have worked together for more than a decade, already resulting in shared research grants, collaborative publications and shared intellectual property. Together, the two institutions will now expand their collaborations to develop new drugs, medical devices, software applications and other novel treatments for cancer, rare diseases and other disorders.
“Joining with Children’s National in the nation’s capital positions Virginia Tech to improve the health and well-being of infants and children around the world,” says Virginia Tech President Tim Sands, Ph.D. “This partnership resonates with our land-grant mission to solve big problems and create new opportunities in Virginia and D.C. through education, technology and research.”
The partnership with Children’s National adds to Virginia Tech’s growing footprint in the Washington D.C. region, which includes plans for a new graduate campus in Alexandria, Va. with a human-centered approach to technological innovation. Sands said the proximity of the two locations – just across the Potomac – will enable researchers to leverage resources, and will also create opportunities with the Virginia Tech campus in Blacksburg, Va. and the Virginia Tech Carilion Health Science and Technology campus in Roanoke, Va.
Carilion Clinic and Children’s National have an existing collaboration for provision of certain specialized pediatric clinical services. The more formalized partnership between Virginia Tech and Children’s National will drive the already strong Virginia Tech-Carilion Clinic partnership, particularly for children’s health initiatives and facilitate collaborations between all three institutions in the pediatric research and clinical service domains.
Children’s National and Virginia Tech will engage in joint faculty recruiting, joint intellectual property, joint training of students and fellows, and collaborative research projects and programs according to Michael Friedlander, Ph.D., Virginia Tech’s vice president for health sciences and technology, and executive director of the Fralin Biomedical Research Institute at VTC.
“The expansion and formalization of our partnership with Children’s National is extremely timely and vital for pediatric research innovation and for translating these innovations into practice to prevent, treat and ultimately cure nervous system cancer in children,” says Friedlander, who has collaborated with Children’s National leaders and researchers for more than 20 years. “Both Virginia Tech and Children’s National have similar values and cultures with a firm commitment to discovery and innovation in the service of society.”
“Brain and other nervous system cancers are among the most common cancers in children (alongside leukemia),” says Friedlander. “With our strength in neurobiology including adult brain cancer research in both humans and companion animals at Virginia Tech and the strength of Children’s National research in pediatric cancer, developmental neuroscience and intellectual disabilities, this is a perfect match.”
The design of the Children’s National Research & Innovation Campus not only makes it conducive for the hospital to strengthen its prestigious partnerships with Virginia Tech and Johnson & Johnson, it also fosters synergies with federal agencies like the Biomedical Advanced Research and Development Authority, which will collaborate with JLABS @ Washington, DC to establish a specialized innovation zone to develop responses to health security threats. As more partners sign on, this convergence of key public and private institutions will accelerate discoveries and bring them to market faster for the benefit of children and adults.
“The Children’s National Research & Innovation Campus pairs an inspirational mission to find new treatments for childhood illness and disease with the ideal environment for early stage companies. I am confident the campus will be a magnet for big ideas and will be an economic boost for Washington DC and the region,” says Jeff Zients, who was appointed chair of the Children’s National Board of Directors effective October 1, 2019. As a CEO and the former director of President Obama’s National Economic Council, Zients says that “When you bring together business, academia, health care and government in the right setting, you create a hotbed for innovation.”
Ranked 7th in National Institutes of Health research funding among pediatric hospitals, Children’s National continues to foster collaborations as it prepares to open its first 158,000-square-foot phase of its Research & Innovation Campus. These key partnerships will enable the hospital to fulfill its mission of keeping children top of mind for healthcare innovation and research while also contributing to Washington D.C.’s thriving innovation economy.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2019/12/VT_CNRICampus_Rendering-sm.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2019-12-11 10:18:202023-07-03 10:40:18Children’s National welcomes Virginia Tech to its new campus
On Nov. 10, 2019, more than 30 pediatric endocrine physicians and nurse practitioners from Washington, D.C., Maryland and Northern Virginia gathered at Children’s National Hospital to discuss the latest in pediatric endocrinology research.
Organized by Paul Kaplowitz, M.D., Professor Emeritus of Pediatrics, this was the third regional pediatric endocrinology meeting since 2012 and the second held at the hospital. “The meetings are a great opportunity for providers to meet regional colleagues who they may communicate with about patients but rarely see face to face,” explains Dr. Kaplowitz.
The providers spent half a day at Children’s National viewing presentations and connecting with their colleagues. Among the presentations was a talk by new Children’s National faculty member Brynn Marks, M.D., MSHPEd, titled, “Medical Education in Diabetes Technologies.”
The presentation highlighted Dr. Marks’ research on how to best teach providers to make optimal use of the information provided by continuous blood glucose monitoring, as well as how to adjust insulin pump settings based on frequent blood glucose testing.
Another notable presentation was by Richard Kahn, Ph.D., recently retired former chief scientific and medical officer at the American Diabetes Association. Dr. Kahn’s talk was titled “Prediabetes: Is it a meaningful diagnosis?”
“This was an excellent talk whose message was that making a diagnosis of ‘prediabetes’ may not be nearly as helpful as we thought, since most patients tests either revert to normal or remain borderline, and there is no treatment or lifestyle change which greatly reduces progression to type 2 diabetes,” says Dr. Kaplowitz.
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Children’s National regional pediatric endocrinology meeting presentations
Welcome from Paul Kaplowitz, M.D., and Children’s National Endocrinology Division Chief Andrew Dauber, M.D.
“Prediabetes: Is it a meaningful diagnosis?”
Richard Kahn, Ph.D., University of North Carolina at Chapel Hill
“Overlapping genetic architecture of Type 2 diabetes and Cystic fibrosis-related diabetes”
Scott Blackman, M.D., Ph.D., Johns Hopkins Medicine
“Pediatric Pituitary Tumors: What we have learned from the NIH cohort”
Christina Tatsi, M.D., Ph.D., National Institutes of Health
“Medical Education in Diabetes Technologies”
Brynn Marks, M.D., MSHPEd, Children’s National Hospital
“A phenotypic female infant with bilateral palpable gonads”
Cortney Bleach, M.D., Walter Reed National Military Medical Center
“Estimating plasma glucose with the FreeStyle Libre Pro CGM in youth: An accuracy analysis”
Miranda Broadney, M.D., MPH, University of Maryland School of Medicine
“Recruiting for research project on “Arginine-Stimulated Copeptin in the diagnosis of central diabetes insipidus”
Chelsi Flippo, M.D., Fellow, National Institutes of Health
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PKD is a genetic disorder characterized by clusters of fluid-filled sacs (cysts) multiplying and interfering with the kidneys’ ability to filter waste from the blood.
When Lisa M. Guay-Woodford, M.D., McGehee Joyce Professor of Pediatrics at Children’s National Hospital, considers a brand-new gift, she likens it to 6 million gallons of “rocket fuel” that will power new research to better understand polycystic kidney disease.
Dr. Guay-Woodford received a $5.7 million dollar gift to support PKD clinical and research activities. PKD is a genetic disorder characterized by clusters of fluid-filled sacs (cysts) multiplying and interfering with the kidneys’ ability to filter waste from the blood. The kidneys’ smooth surface transforms to a bumpy texture as the essential organs grow oversized and riddled with cysts.
The extraordinary generosity got its start in an ordinary clinical visit.
Dr. Guay-Woodford saw a young patient in her clinic at Children’s National a few times in 2015. The child’s diagnosis sparked a voyage of discovery for the patient’s extended family and, ultimately, they attended a presentation she gave during a regional meeting about PKD. That led to a telephone conversation and in-person meeting as they invited her to describe “the white space” between what was being done at the time to better understand PKD and what could be done.
“It’s the power of the art and science of medicine. They come to see people like me because of the science. If we can convey to patients and families that who they are and their unique concerns are really important to researchers, that becomes a powerful connection,” she says. “The art plus the science equals hope. That is what these families are looking for: We give people the latest insights about their disease because information is power.”
The infusion of new funding will strengthen the global initiative’s four pillars:
Coordinated care for children and families impacted by renal cystic disease. The Inherited and Polycystic Kidney Disease (IPKD) program, launched September 2019, includes a cadre of experts working together as a team in the medical home so that “in a single, one-stop visit, Children’s National can address the myriad concerns they have,” she explains. A multi-disciplinary team that includes nephrologists, hepatologists and endocrinology experts meets weekly to ensure the Center of Excellence provides the highest-caliber patient care. The team includes genetic counselors to empower families with knowledge about genetic risks and testing opportunities. A nurse helps families navigate the maze of who to call about which issue. Psychologists help to ease anxiety. “There is stress. There is fear. There is pain that can be associated with this set of diseases. The good news is we can control their medical issues. The bad news is some children have difficulty coping. Our psychologists help children cope so they can be a child and do the normal things that children do,” she says.
Strengthening global databases to capture PKD variations. The team will expand its outreach to other centers located around the world – including Australia, Europe, India and Latin America – caring for patients with both the recessive and dominant forms of polycystic kidney disease, to better understand the variety of ways the disease can manifest in children. “We really don’t know a lot about kids with the dominant form of the disease. How hard should we push to control their blood pressure, knowing that could ease symptoms? What are the ramifications of experiencing acute pain compared with chronic pain? How much do these pain flareups interfere with daily life and a child’s sense of self,” she asks. Capturing the nuances of the worldwide experience offers the power of harnessing even more data. And ensuring that teams collect data in a consistent way means each group would have the potential to extract the most useful information from database queries.
Filling a ‘desperate need’ for biomarkers. Developing clinical trials for new therapies requires having biomarkers that indicate the disease course. Such biomarkers have been instrumental in personalizing care for patients with other chronic conditions. “We are in desperate need for such biomarkers, and this new funding will underwrite pilot studies to identify and validate these disease markers. The first bite at the apple will leverage our imaging data to identify promising biomarkers,” she says.
Genetic mechanisms that trigger kidney disease. About 500,000 people in the U.S. have PKD. In many cases, children inherit a genetic mutation but, often, their genetic mutation develops spontaneously. Dr. Guay-Woodford’s research about the mechanisms that make certain inherited renal disorders lethal, such as autosomal recessive polycystic kidney disease, is recognized around the world. The fourth pillar of the new project provides funding to continue her lab’s research efforts to improve the mechanistic understanding of what triggers PKD.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2019/11/polycystic-kidney-disease.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2019-11-14 17:30:452023-05-01 10:03:46$6M gift powers new PKD clinical and research activities
“We know that bariatric surgery improves markers of kidney health in severely obese adults and adolescents,” says Marva Moxey-Mims, M.D. “This research helps to elucidate possible differences in kidney disease outcomes between children and adults post-surgery.”
Adolescents with Type 2 diabetes experienced more hyperfiltration and earlier attenuation of their elevated urine albumin-to-creatinine ratio (UACR) after gastric bypass surgery compared with adults. This finding contrasts with adolescents or adults who did not have diabetes prior to surgery, according to research presented Nov. 8, 2019, during the American Society of Nephrology’s Kidney Week 2019, the world’s largest gathering of kidney researchers.
“Findings from this work support a recent policy statement by the American Academy of Pediatrics (AAP) that advocates for increasing severely obese youths’ access to bariatric surgery,” says Marva Moxey-Mims, M.D., Chief of the Division of Nephrology at Children’s National Hospital and a study co-author. “We know that bariatric surgery improves markers of kidney health in severely obese adults and adolescents. This research helps to elucidate possible differences in kidney disease outcomes between children and adults post-surgery.”
According to the AAP, the prevalence of severe obesity in youth aged 12 to 19 has nearly doubled since 1999. Now, 4.5 million U.S. children are affected by severe obesity, defined as having a body mass index ≥35 or ≥120% of the 95th percentile for age and sex.
In a Roux-en-Y gastric bypass, the surgeon staples the stomach to make it smaller, so people eat less. Then, they attach the lower part of the small intestine in a way that bypasses most of the stomach so the body takes in fewer calories.
The multi-institutional study team examined the health effects of such gastric bypass surgeries by comparing 161 adolescents with 396 adults enrolled in related studies. They compared their estimated glomerular filtration rates by serum creatinine and cystatin C. UACR was also compared at various time periods, up till five years after surgery.
Across the board, adolescents had higher UACR – a key marker for chronic kidney disease – than adults. However, for kids who had Type 2 diabetes prior to surgery, the prevalence of elevated UACR levels dip from 29% pre-surgery to 6% one year post-surgery. By contrast, adults who had diabetes prior to surgery and elevated UACR did not see a significant reduction in UACR until five years post-surgery.
While hyperfiltration prevalence was similar in study participants who did not have Type 2 diabetes, adolescents who had Type 2 diabetes prior to surgery had an increased prevalence of hyperfiltration for the duration of the study period.
Financial support for research described in this post was provided by the National Institute of Diabetes and Digestive and Kidney Diseases.
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ASN Kidney Week 2019 presentation
“Five-year kidney outcomes of bariatric surgery in adolescents compared with adults”
Friday, Nov. 8, 2019, 10 a.m. to noon (EST)
Petter Bjornstad, University of Colorado School of Medicine; Todd Jenkins, Edward Nehus and Mark Mitsnefes, all of Cincinnati Children’s Hospital; Marva M. Moxey-Mims, Children’s National Hospital; and Thomas H. Inge, Children’s Hospital Colorado.
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Morphometric and textural analyses of magnetic resonance imaging can point out subtle architectural deviations associated with fetal growth restriction during the second half of pregnancy, a first-time finding that has the promise to lead to earlier intervention.
Morphometric and textural analyses of magnetic resonance imaging (MRI) can point out subtle architectural deviations that are associated with fetal growth restriction (FGR) during the second half of pregnancy. The first-time finding hints at the potential to spot otherwise hidden placental woes earlier and intervene in a more timely fashion, a research team led by Children’s National Hospital faculty reports in Pediatric Research.
“We found reduced placental size, as expected, but also determined that the textural metrics are accelerated in FGR when factoring in gestational age, suggesting premature placental aging in FGR,” says Nickie Andescavage, M.D., a neonatologist at Children’s National and the study’s lead author. “While morphometric and textural features can discriminate placental differences between FGR cases with and without Doppler abnormalities, the pattern of affected features differs between these sub-groups. Of note, placental insufficiency with abnormal Doppler findings have significant differences in the signal-intensity metrics, perhaps related to differences of water content within the placenta.”
The placenta, an organ shared by the pregnant woman and the developing fetus, delivers oxygen and nutrients to the developing fetus and ferries away waste products. Placental insufficiency is characterized by a placenta that develops poorly or is damaged, impairing blood flow, and can result in still birth or death shortly after birth. Surviving infants may be born preterm or suffer early brain injury; later in life, they may experience cardiovascular, metabolic or neuropsychiatric problems.
Because there are no available tools to help clinicians identify small but critical changes in placental architecture during pregnancy, placental insufficiency often is found after some damage is already done. Typically, it is discovered when FGR is diagnosed, when a fetus weighs less than 9 of 10 fetuses of the same gestational age.
“There is a growing appreciation for the prenatal origin of some neuropsychiatric disorders that manifest years to decades later. Those nine months of gestation very much define the breath of who we later become as adults,” says Catherine Limperopoulos, Ph.D., director of MRI Research of the Developing Brain at Children’s National and the study’s senior author. “By identifying better biomarkers of fetal distress at an earlier stage in pregnancy and refining our imaging toolkit to detect them, we set the stage to be able to intervene earlier and improve children’s overall outcomes.”
The research team studied 32 healthy pregnancies and compared them with 34 pregnancies complicated by FGR. These women underwent up to two MRIs between 20 weeks to 40 weeks gestation. They also had abdominal circumference, fetal head circumference and fetal femur length measured as well as fetal weight estimated.
In pregnancies complicated by FGR, placentas were smaller, thinner and shorter than uncomplicated pregnancies and had decreased placental volume. Ten of 13 textural and morphometric features that differed between the two groups were associated with absolute birth weight.
“Interestingly, when FGR is diagnosed in the second trimester, placental volume, elongation and thickness are significantly reduced compared with healthy pregnancies, whereas the late-onset of FGR only affects placental volume,” Limperopoulos adds. “We believe with early-onset FGR there is a more significant reduction in the developing placental units that is detected by gross measures of size and shape. By the third trimester, the overall shape of the placenta seems to have been well defined so that primarily volume is affected in late-onset FGR.”
In addition to Dr. Andescavage and Limperopoulos, study co-authors include Sonia Dahdouh, Sayali Yewale, Dorothy Bulas, M.D., chief of the Division of Diagnostic Imaging and Radiology, and Biostatistician, Marni Jacobs, Ph.D., MPH, all of Children’s National; Sara Iqbal, of MedStar Washington Hospital Center; and Ahmet Baschat, of Johns Hopkins Center for Fetal Therapy.
Financial support for research described in this post was provided by the National Institutes of Health under award number 1U54HD090257, R01-HL116585, UL1TR000075 and KL2TR000076, and the Clinical-Translational Science Institute-Children’s National.
