To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular ...

The 16-year-old’s infectious smile and easy charm have won over everyone from community members to politicians in both Indianapolis and Washington, D.C. But behind his charisma is a powerful cause: a ...

The purpose of this report is to re-evaluate such a therapeutic program in muscular dystrophy when the drugs are used singly, in combination and with or without exercise. Objective serial ...

The committee was also scheduled to vote on including Duchenne muscular dystrophy. On April 17 ... a professor for genetics and metabolism in the pediatrics department at the University of ...

In response, the federal government tasked the American College of Medical Genetics (ACMG ... metachromatic leukodystrophy and Duchenne muscular dystrophy for the RUSP . The disbanding of ...

In addition, we expect to share early clinical results later this year from both BMN 351 for Duchenne Muscular Dystrophy and BMN ... American College of Medical Genetics and Genomics meeting ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular ...

to treat Duchenne muscular dystrophy (DMD) in patients aged 6 years and older who can walk. Conditional marketing authorization is granted when the benefit of immediate availability outweighs the ...