Research Advances

Blog Post

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

By Jeanene Swanson | November 15, 2019
Blog Post

NS Pharma Submits New Drug Application to FDA for Viltolarsen to Treat DMD Amenable to Exon 53 Skipping

By Jeanene Swanson | October 7, 2019
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Blog Post

In a Letter to the Duchenne Community, Santhera Announces Positive Results of Phase 2 Vamorolone Study, EMA’s Review of Puldysa to Treat Symptoms of DMD

By MDA Staff | September 26, 2019
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Blog Post

FDA Approves AveXis’ Zolgensma for Treatment of Spinal Muscular Atrophy in Pediatric Patients

By Jeanene Swanson | May 24, 2019
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Do You Know ALS? Read About the Latest Research.

By Jeanene Swanson | May 15, 2019
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Biogen Releases Letter to ALS Community

By MDA Staff | May 2, 2019
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FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease

By Jeanene Swanson | March 7, 2019
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FDA Accepts Sarepta Therapeutics’ New Drug Application (NDA) for Golodirsen to Treat DMD Amenable to Exon 53 Skipping

By Jeanene Swanson | March 1, 2019
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MDA Awards Venture Philanthropy Grant to AcuraStem to Develop Treatment for ALS

By Jeanene Swanson | January 15, 2019
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FDA Grants Orphan Drug Designation to Myonexus Therapeutics’ MYO-102 Gene Therapy Drug for Limb-Girdle Muscular Dystrophy Type 2D

By Jeanene Swanson | January 4, 2019