US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

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Spinal Muscular Atrophy Added to National List of Disorders to Screen Newborns

Landmark decision for the SMA community recognizes importance of early diagnosis

CHICAGO, July 5, 2018 – The Muscular Dystrophy Association commends Secretary Azar on adding Spinal Muscular Atrophy (SMA) to the Recommended Uniform Screening Panel (RUSP).

Adding SMA to the list will help ensure that babies born with SMA are identified, so that they have the opportunity to benefit from early treatment and intervention. This testing can also provide families with a genetic diagnosis — information that often is required to determine whether their child is eligible to participate in clinical trials.

MDA is an umbrella organization representing more than 40 different disorders – including SMA. We believe that both our 150 MDA Care Centers across the U.S., and MOVR (our data hub) can play a key role in confirmatory diagnoses, treatment, long-term care and follow up.

To learn more about newborn screening and its impact on the neuromuscular community, read a Q&A with newborn screening expert Dr. Rodney Howell, who also serves as Chairman of MDA’s Board of Directors. You can also read a blog about the importance of this decision to the SMA and MDA community here.