September 2026

Adults with SMA Share Their Unmet Needs and Independence Barriers in Washington D.C.

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This week, the unmet health and everyday living needs of adults with spinal muscular atrophy (SMA) took center stage in Washington, DC, in an event organized by Cure SMA. Adults with SMA from across the country participated in the September 27-28 event to discuss the significant challenges and barriers they face that impact their health

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The 2026 Annual SMA Research & Clinical Care Meeting Summaries Are Here!

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In June, SMA researchers and clinicians from around the world gathered in Orlando for the 30th Annual SMA Research & Clinical Care Meeting — to share early findings, compare notes across disciplines, and drive continued progress in clinical care and new treatments for people living with SMA. This year, we’re doing something new: all the

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Scholar Rock Receives Approval for ISEMBYLD™ for the Treatment of SMA

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Scholar Rock  today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for ISEMBYLD™ (apitegromab-mstn) to treat spinal muscular atrophy (SMA) in adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. This approval brings the first muscle-targeted treatment to

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Genentech Shares Highlights from an Unforgettable Week at the Annual SMA Conference

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From a lively “Evryday Café” exhibit hall experience to an advocacy reception featuring conversations on policy and access, Genentech recaps a full week connecting with the SMA community at this year’s Annual SMA Conference. Read their latest community update here.

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