September 2023

Identifying Biomarkers of Spinal Muscular Atrophy for Further Development

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An important part of Cure SMA’s core mission is to carry out and support research that will improve the quality of life for all people with SMA. We recently published a new research article, “Identifying Biomarkers of Spinal Muscular Atrophy for Further Development.” This article appeared in the July 12, 2023 issue of the peer-reviewed […]

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NMD Pharma Initiates Phase II Trial for SMA

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NMD Pharma, a clinical-stage biotech company leading the development of novel first-in-class therapies for severe neuromuscular disorders, recently announced that the first SMA patient has been dosed in their Phase II clinical trial of the CIC-1 inhibitor NMD670. To read the full press release, visit this link. MD670 is a first-in-class small molecule inhibitor of

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Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial

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Today, Scholar Rock announced that enrollment in their Phase 3 SAPPHIRE trial is now complete. The Phase 3 trial will evaluate the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) who are receiving SMN therapy (either nusinersen or risdiplam). Scholar Rock also announced that it plans to

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Biohaven Completes Enrollment in Pivotal Phase 3 Study

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Recently, Biohaven released a statement on the completed enrollment of their pivotal Phase 3 Study of Taldefgrobep Alfa in people with SMA. Check out Biohaven’s official press release here. About Taldefgrobep and the Phase 3 Trial Taldefgrobep is an investigational, muscle-targeted recombinant protein with the potential to enhance muscle mass and strength in people living

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The State of SMA – Toward Improved Therapeutics and Care

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Each year, Cure SMA collaborates with our Scientific Advisory Board to plan a Special Session that will be held during the Annual Research & Clinical Care Meeting. This Special Session is designed to focus on a topic that is currently of great interest to both researchers and clinicians. This year’s Special Session, “The State of

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