Author Archives: Marisa Wexler MS

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…

Sarepta Therapeutics‘ investigational gene therapy SRP-9001 showed early signs of biologic efficacy in muscle biopsies from 11 boys with Duchenne muscular dystrophy (DMD) treated in a Phase 1 clinical trial, the company announced in a press release. “Robust” expression of micro-dystrophin was evident in cells at 12…

Two more participants have been dosed in the IGNITE DMD clinical trial, bringing to eight the total number of Duchenne muscular dystrophy (DMD) patients thus far given Solid Biosciences‘ investigational gene therapy SGT-001. The U.S. trial is testing SGT-001’s safety and efficacy in boys with DMD ages…

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…

Jesse’s Journey, a Canadian nonprofit founded by parents of a child with Duchenne muscular dystrophy (DMD), has given a total CA$1.7 million to support four projects looking to protect muscle fibers, or treat patients through gene editing and stem cells approaches. The award, worth about $1.4 million, is…

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…

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. Women with limb girdle muscular dystrophy type R9 (LGMDR9) are not at a greater risk of pregnancy complications,…

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. Once-weekly corticosteroid treatment was safe and well-tolerated in people with Becker muscular dystrophy (BMD) or limb-girdle muscular dystrophy…

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…

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,…