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The company was testing its drug, losmapimod, in 260 patients with facioscapulohumeral muscular dystrophy (FSHD) who received either the therapy or a placebo for 48 weeks. The study also failed to ...
The FSHD Society (named "FSH Society" until 2019) [170] was founded in 1991 on the East Coast by two individuals with FSHD, Daniel Perez and Stephen Jacobsen. [171] [172] The FSHD Society claims to have advocated for the standardization of the disease name facioscapulohumeral muscular dystrophy and its abbreviation FSHD. [170]
Nila Morton was born with a rare form of muscular dystrophy called Ullrich, which requires her to rely on a wheelchair. When it came time to travel home for the holidays this year, she at first ...
The diagnosis of muscular dystrophy is based on the results of muscle biopsy, increased creatine phosphokinase (CpK3), electromyography, and genetic testing. A physical examination and the patient's medical history will help the doctor determine the type of muscular dystrophy.
Toshifumi (Toshi) Yokota (Japanese: 横田俊文, romanized: Yokota Toshifumi) is a biomedical scientist and professor of medical genetics at the University of Alberta, holding the titles of the Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair and the Henri M. Toupin Chair in Neurological Science. [1]
(Reuters) -A young patient died due to cardiac arrest after receiving Pfizer's experimental gene therapy being tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular ...
Wilton's group was the first to report specific exon skipping in an animal model of Duchenne muscular dystrophy, which led ultimately to the development of a complete panel of splice-switching oligonucleotides. [31] Their work demonstrated the efficacy of phosphorodiamidate morpholino oligomers (PMOs) as splice-switching agents. [5]
(Reuters) -Sarepta Therapeutics' gene therapy to treat Duchenne muscular dystrophy (DMD), a progressive muscle-wasting disorder, failed to meet the main goal of a late-stage trial when tested in ...