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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Amylyx Pharmaceuticals Announces Positive Results from Phase 2 Study of AMX0035 for Treatment of ALS

By Jeanene Swanson | December 19, 2019
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Santhera Pharmaceuticals Announces Publication of Positive Long-Term Results from Retrospective Study of Idebenone in DMD

By Jeanene Swanson | December 18, 2019
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Solid Biosciences Releases Letter to DMD Community Announcing Update on IGNITE DMD Trial, Resolution of Serious Adverse Event

By MDA Staff | December 18, 2019
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Wave Life Sciences Releases Letter to DMD Community Announcing Discontinuation of Clinical Trials Program for Therapies Amenable to Skipping Exons 51 and 53

By MDA Staff | December 16, 2019
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New MDA Educational Programs for Clinicians Offer a Detailed View on Selected Topics in Neuromuscular Disease

By MDA Staff | December 13, 2019
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FDA Approves Sarepta Therapeutics’ Vyondys 53 for Treatment of DMD Amenable to Exon 53 Skipping

By Jeanene Swanson | December 13, 2019
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FDA Approves Aquestive Therapeutics’ Exservan for the Treatment of ALS

By Jeanene Swanson | December 6, 2019
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Kadimastem Announces Positive Interim Results from Phase 1/2a Study of AstroRx in ALS

By Jeanene Swanson | December 5, 2019
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FDA Accepts Roche Genentech’s NDA for Risdiplam for the Treatment of SMA

By Jeanene Swanson | December 3, 2019
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Zogenix Announces Positive Results from Pivotal Study of Investigational Treatment MT1621 for TK2 Deficiency

By Jeanene Swanson | December 2, 2019