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,…
Flavocoxid was well-tolerated and lessened inflammation in a small clinical trial involving boys with Duchenne muscular dystrophy (DMD). Researchers said these results support Flavocoxid’s further investigation as a potential DMD therapy. The findings were published in the journal Brain Sciences, in “A Phase 1/2…
Through a program called MDA Engage, the Muscular Dystrophy Association (MDA) is offering virtual educational events for people affected by muscular dystrophy and other neuromuscular diseases. The events are aimed at people living with these conditions, as well as their loved ones. MDA Engage offers three types…
Treatment with Translarna (ataluren) increased levels of the protein dystrophin in young boys with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, new clinical trial data show. The findings were presented in a webcast by Stuart W. Peltz, PhD, the CEO of PTC Therapeutics, which…
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