Author Archives: Marisa Wexler MS

LION-101 Gene Therapy for Rare MD Type Now on FDA Fast Track

The U.S. Food and Drug Administration (FDA) has granted fast track designation to LION-101, an investigational gene therapy that Asklepios BioPharmaceutical (AskBio) is developing as a treatment for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). This designation is given to therapies designed to fill an unmet need in medical care for…

Vita Raises $32M to Advance Cell Therapies for LGMD, Other Dystrophies

Vita Therapeutics announced that it has raised $32 million in financing to advance its work into cell-based therapies for muscular dystrophy. “At Vita Therapeutics our mission is to deliver long-term disease-modifying cell engineered treatments for patients living with muscular dystrophies and other high unmet medical needs,” Douglas Falk, the…

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…