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An advisory committee of the U.S. Department of Health and Human Services is expected to vote this fall on whether to recommend newborns be screened for Duchenne muscular dystrophy, or DMD.
The Advisory Committee on Heritable Disorders in Newborns and Children is slated to meet on Nov. 14 and 15 to decide on whether to add DMD to the HHS’s Recommended Uniform Screening Panel for newborns, according to Endpoints News, which cited Parent Project Muscular Dystrophy, or PPMD.
The parent group, along with the Muscular Dystrophy Association, has requested that the committee delay voting on the issue until later in the year as additional supporting evidence is expected to become available “in the near future.” The HHS committee voted Thursday to delay its decision for up to one year, PPMD said on its website.
Proponents of early screening for DMD maintain that early diagnosis and intervention can help improve patient outcomes.
DMD has become a popular area for drug development in recent years. Companies with DMD therapies either on the market or in development include Sarepta (NASDAQ:SRPT), Pfizer (PFE), Catalyst (NASDAQ:CPRX), Edgewise Therapeutics (EWTX), Satellos (OTC:MSCLF), PepGen (PEPG), Entrada (TRDA), Dyne Therapeutics (DYN), Regenxbio (RGNX), Wave Life Sciences (WVE), Vertex Pharmaceuticals (VRTX), Solid Biosciences (SLDB), Capricor (CAPR), PTC Therapeutics (PTCT), Avidity (RNA), and Roche (OTCQX:RHHBY).
More on Catalyst Pharmaceuticals, Sarepta Therapeutics, etc.
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