Science + Research

Find the latest science and research updates as well as information to help you understand this ever-evolving field.

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New MDA Educational Programs for Clinicians Offer a Detailed View on Selected Topics in Neuromuscular Disease

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FDA Approves Sarepta Therapeutics’ Vyondys 53 for Treatment of DMD Amenable to Exon 53 Skipping

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FDA Approves Aquestive Therapeutics’ Exservan for the Treatment of ALS

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Kadimastem Announces Positive Interim Results from Phase 1/2a Study of AstroRx in ALS

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FDA Accepts Roche Genentech’s NDA for Risdiplam for the Treatment of SMA

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Zogenix Announces Positive Results from Pivotal Study of Investigational Treatment MT1621 for TK2 Deficiency

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Santhera Pharmaceuticals Releases Letter to DMD Community Announcing Temporary Suspension of Enrollment in BreatheDMD Expanded Access Program

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Roche Genentech Announces Positive Results from Pivotal Study of Risdiplam in SMA Types 2 and 3

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Solid Biosciences Releases Letter to DMD Community Announcing Hold on IGNITE DMD Trial Due to a Serious Adverse Event

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Roche Genentech Releases Letter to DMD Community Announcing Discontinuation of Clinical Trials Program for DMD