News

#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 — Amondys 45 Safe and Well-tolerated in Phase 1/2 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. Treatment with Amondys 45 was safe and well-tolerated, showing little to no evidence of accumulation in the blood…