SMA Added to National List of Disorders to Screen for at Birth

After significant collaborative work among many stakeholders in the spinal muscular atrophy (SMA) community, we are pleased to share that the Secretary of Health and Human Services (HHS), Dr. Alex Azar, has accepted the recommendation to add SMA to the Recommended Uniform Screening Panel (RUSP) for newborns. This is a landmark decision for the SMA . . .

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13 New MDA Grants Totaling $2.6 Million Push for More Progress

MDA has announced the award of 13 new grants totaling $2.6 million as part of its continued commitment to finding treatments and cures for the diseases in its program. Each of the 13 new grants funds a project that aims to increase our understanding of the causes or underlying mechanisms of neuromuscular disease or potential . . .

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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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FDA: Translarna is Not Ready for Approval at This Time

PTC Therapeutics reported today the U.S. Food and Drug Administration’s Office of Drug Evaluation is unable to approve the company’s New Drug Application for ataluren (brand name Translarna) for the treatment of Duchenne muscular dystrophy (DMD) caused by a premature stop codon, or nonsense mutation, in its current form.

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Remembering Jerry

After the news of MDA champion Jerry Lewis’s passing broke Sunday, scores of admirers took to social media to remember the comedian and philanthropist. Here are just a few of the thousands of tweets sent by some of the entertainment business’ brightest stars — and some of MDA’s staunchest partners. Want to add your remembrance to the chorus? Visit our Tributes page, where we are collecting memories for Jerry’s family.

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