Gene Therapy

Blog Post

Audentes Therapeutics Announces Plans to Develop Gene-Targeted Therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1

By Jeanene Swanson | April 11, 2019
Blog Post

Muscular Dystrophy Association Awards Eight Grants Totaling More Than $2 Million for ALS Research

By Jeanene Swanson | April 8, 2019
Blog Post

Sarepta Therapeutics Announces Positive Interim Results from Study of Casimersen to Treat DMD Amenable to Exon 45 Skipping

By Jeanene Swanson | April 4, 2019
Blog Post

Sarepta Therapeutics Announces Positive Interim Results in Gene Therapy Trial for LGMD2E and Acquisition of Myonexus Therapeutics

By Jeanene Swanson | March 15, 2019
Blog Post

CMTA and MDA Co-Fund Proof-of-Concept Study Using AAV Vector to Deliver Gene Replacement Therapy in X-linked Charcot-Marie-Tooth Disease

By Jeanene Swanson | March 1, 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

Solid Biosciences Announces Preliminary Results of DMD Gene Therapy Trial

By Jeanene Swanson | February 8, 2019
Blog Post

Wave Life Sciences Announces Phase 2/3 Duchenne Muscular Dystrophy Clinical Trial of Suvodirsen (WVE-210201) Selected for FDA Complex Innovative Trial Designs Pilot Program

By Jeanene Swanson | January 8, 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