Author Archives: Marisa Wexler MS

Gene Therapy Trial for Rare Form of MD Planned for Next Year

Asklepios BioPharmaceutical (AskBio) is planning to launch a Phase 1/2 clinical study to evaluate LION-101, its investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Dosing in the trial is expected to start in the first half of next year. “We are excited to begin clinical trials…

FDA Puts Investigational FSHD Therapy Losmapimod on Fast Track

The U.S. Food and Drug Administration (FDA) has granted fast track designation to losmapimod, a potential treatment for facioscapulohumeral muscular dystrophy (FSHD) being developed by Fulcrum Therapeutics. The designation is intended to help bring effective treatments for serious conditions to market more quickly. It grants Fulcrum, as…

#AANAM – Presenter Compares Features of DM1 and DM2

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. People with myotonic dystrophy type 2 (DM2) tend to be older at symptom onset than those with myotonic…

#AANAM – Investigational DM1 Therapy Shows Promise

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. An investigational therapy called AOC-1001 decreased levels of the toxic RNA molecule that causes…

FDA Places Pamrevlumab for DMD on Fast Track

The U.S. Food and Drug Administration has granted fast track designation to pamrevlumab, an investigational treatment for Duchenne muscular dystrophy (DMD). The designation is given to medications that have the potential to treat serious conditions and to fill an unmet medical need. The designation allows the therapy’s developer, FibroGen,…