Research Advances

Featured Article

A Good Beginning: Newborn Screening

By Donna Albrecht | January 17, 2020
Blog Post

FDA Approves ITF Pharma’s Tiglutik via PEG Tube for the Treatment of ALS

By Jeanene Swanson | December 20, 2019
Blog Post

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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Blog Post

Solid Biosciences Releases Letter to DMD Community Announcing Update on IGNITE DMD Trial, Resolution of Serious Adverse Event

By MDA Staff | December 18, 2019
Blog Post

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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Blog Post

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

By Jeanene Swanson | December 13, 2019
Blog Post

FDA Approves Aquestive Therapeutics’ Exservan for the Treatment of ALS

By Jeanene Swanson | December 6, 2019
Blog Post

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

By Jeanene Swanson | December 5, 2019
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Blog Post

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

By Jeanene Swanson | December 3, 2019
Blog Post

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

By Jeanene Swanson | December 2, 2019