Research Advances

Blog Post

Biogen’s Early Access Program for Tofersen is Now Open for Individuals with Rapidly Progressing SOD1-ALS

By Sujatha Gurunathan | July 6, 2021
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How Viral Vectors Deliver the Goods in Gene Therapy

By Larry Luxner | May 14, 2021
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New Research Channels for Myotonia Congenita

By MDA Staff | February 23, 2021
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The Search for Inclusion Body Myositis Treatment

By MDA Staff | December 4, 2020
Blog Post

Once a Wild Idea, Successful First-Generation Exon-Skipping Therapies Pave the Way for Personalized Treatments

By Sujatha Gurunathan | November 17, 2020
Blog Post

Scholar Rock Announces Positive Results from Interim Analysis of TOPAZ Phase 2 Study of SRK-015 to Treat SMA

By Sujatha Gurunathan | October 28, 2020
Blog Post

Amylyx Announces More Results from Study of AMX0035 to Treat ALS

By Sujatha Gurunathan | October 19, 2020
Blog Post

FDA Accepts Sarepta Therapeutics’ New Drug Application for DMD Treatment Casimersen

By Sujatha Gurunathan | August 26, 2020
Featured Article

The Expanding Therapeutic Landscape for Myasthenia Gravis

By MDA Staff | August 14, 2020
Blog Post

Biogen Announces Positive Results from Phase 1/2 Study of Tofersen to Treat a Genetic Form of ALS

By Sujatha Gurunathan | July 14, 2020