MDA 2021

#MDA2021 – Long-term Vamorolone Slowed Disease Progression in DMD Boys

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Long-term treatment with the experimental steroid therapy vamorolone safely and effectively delays motor function decline in boys with…

#MDA2021 – DMD Gene Therapy PF-06939926 Continues to Show Promise

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The investigational gene therapy PF-06939926 continues to demonstrate an acceptable safety profile and the potential for…

#MDA2021 – Gene Therapy SGT-001 Shows Continued Benefit in DMD Boys

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The investigational gene therapy SGT-001 continues to lead to improvements in physical functioning, disease-related biomarkers, and patient-reported outcomes…

#MDA2021 — Gene Therapy for DMD Exon Duplication Showing Promise in Trial

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Audentes Therapeutics’ experimental exon skipping gene therapy may safely and effectively increase dystrophin levels and stabilize muscle function…

#MDA2021 – SRP-5051 Shows Promise in Animal Studies, Supporting Phase 2 Trial

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Monthly administration of SRP-5051 — Sarepta Therapeutics’ investigational, potentially improved version of its Exondys 51 (eteplirsen) therapy…