Research Advances

Blog Post

FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease

By Jeanene Swanson | March 7, 2019
Blog Post

FDA Accepts Sarepta Therapeutics’ New Drug Application (NDA) for Golodirsen to Treat DMD Amenable to Exon 53 Skipping

By Jeanene Swanson | March 1, 2019
Blog Post

MDA Awards Venture Philanthropy Grant to AcuraStem to Develop Treatment for ALS

By Jeanene Swanson | January 15, 2019
Blog Post

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
DNA strand and Cancer Cell Concept Science or medical background, 3d illustration.
Blog Post

C9ORF72 Mutation Most Common Cause of Familial ALS, FTD, ALS-FTD

By Amy Madsen | September 20, 2011
Woman doctor using tablet computer in hospital
Blog Post

National ALS Registry Now Open

By Miriam Davidson | October 19, 2010