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Children’s National Health System named as member of the Parent Project Muscular Dystrophy’s (PPMD) Certified Duchenne Care Centers

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Children’s National Health System is now part of a growing Duchenne care network, becoming the newest member of the Parent Project Muscular Dystrophy’s (PPMD) Certified Duchenne Care Program.

The certification process to become a Certified Duchenne Care Center (CDCC) was grounded in the idea that comprehensive Duchenne care and services should be available and accessible to as many families as possible. By joining the network of PPMD Certified Duchenne Care Centers and standardizing care, Children’s National’s Neuromuscular Medicine Program is also improving Duchenne research and clinical trials by decreasing variability in care and increasing the quality of clinical trial outcome measures. This results in accelerating the time it takes therapies to reach the patients who need them.

By allowing neuromuscular patients of all diagnoses access to the comprehensive teams of sub-specialists serving the Duchenne population, Children’s National and other PPMD Certified Duchenne Care Centers will improve the care of all patients with neuromuscular diagnoses.

Muscular Dystrophy Association awards grants to two Children’s National scientists

Marshall Hogarth, Ph.D

Marshall Hogarth, Ph.D

James Novak, Ph.D.

James Novak, Ph.D.

Two Children’s National Health System research scientists, Marshall Hogarth, Ph.D. and James Novak, Ph.D., have received Post-Doctoral Development Grants from the Muscular Dystrophy Association (MDA) as part of funding awarded to young, rising researchers who are poised to become independent investigators.

Over the next three years, Hogarth and Novak will be allotted $180,000 each to underwrite their individual research projects.

Hogarth’s research is focused on limb-girdle muscular dystrophy (LGMD), a disease which presents as muscle weakness when patients are in their late teens before rapidly progressing to severe debilitation. The MDA grant will allow Hogarth to continue his research investigating the replacement of muscle with fatty tissue and the role this plays in the late onset and subsequent progression of LGMD in patients.

Novak focuses mainly on researching Duchenne Muscular Dystrophy (DMD), a severely debilitating form of MD, that leads to progressive muscle weakness and respiratory and cardiac failure. Currently, the only Food and  Drug Administration (FDA)  approved treatment for DMD is exon-skipping. The MDA grant will support Novak’s study of the mechanisms that regulate the delivery of exon-skipping drugs in muscle, in order to identify new therapeutic targets and improve drug efficacy for patients with DMD.

While Hogarth and Novak focus on different aspects of neuromuscular disease, both look forward to making significant contributions that lead to overall improvements in the treatment of patients impacted by muscular dystrophy.