Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

Newborn Screening in the U.S. is One Step Closer to Including SMA

The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), a federal committee that oversees newborn screening, voted today to add spinal muscular atrophy (SMA) to the national list of disorders recommended for screening at birth. Following today’s vote, the Secretary of Health and Human Services (HHS) will make a final decision on whether . . .

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Recommendation for SMA to be added to RUSP is announced

Today, the Advisory Committee on Heritable Disorders in Newborns and Children voted to recommend to the Secretary of Health and Human Services that spinal muscular atrophy (SMA) be added to the Recommended Uniform Screening Panel (RUSP), the national list that guides states on which diseases should be tested for at birth. Newborn screening is essential . . .

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Ezutromid Shows Potential to Treat DMD

Ezutromid

Summit Therapeutics plc has announced encouraging interim results from its phase 2 PhaseOut DMD clinical trial that suggest the MDA research grant-funded experimental treatment ezutromid (formerly SMT C1100) potentially may be effective as a treatment for DMD.

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