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Facioscapulohumeral muscular dystrophy ( FSHD) is a type of muscular dystrophy, a group of heritable diseases that cause degeneration of muscle and progressive weakness. Per the name, FSHD tends to sequentially weaken the muscles of the face, those that position the scapula, and those overlying the humerus bone of the upper arm.
Last year, the drug – Elevidys, from the biotech company Sarepta Therapeutics – was approved to treat only children ages 4 and 5 with Duchenne muscular dystrophy, one of the most severe forms ...
By Leroy Leo and Sriparna Roy. (Reuters) -The U.S. Food and Drug Administration allowed the expanded use of Sarepta Therapeutics' gene therapy for patients with Duchenne muscular dystrophy aged ...
In February 2019, Sarepta acquired five gene therapy candidates for $165 million after one of them, MYO-101, produced results with a new gene therapy candidate for patients with Limb-Girdle muscular dystrophy; two months after receiving a single treatment, muscles from all three patients were producing the protein they couldn't make on their own.
Losmapimod ( GW856553X) is an investigational drug being developed by Fulcrum Therapeutics for the treatment of facioscapulohumeral muscular dystrophy (FSHD); a phase III clinical trial is pending approval. Losmapimod selectively inhibits enzymes p38α/β mitogen-activated protein kinases (MAPKs), which are modulators of DUX4 expression and ...
(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 ...
Duchenne muscular dystrophy ( DMD) is a severe type of muscular dystrophy predominantly affecting boys. [3] [6] [7] The onset of muscle weakness typically begins around age four, with rapid progression. [2] Initially, muscle loss occurs in the thighs and pelvis, extending to the arms, [3] which can lead to difficulties in standing up. [3]
The case for Sarepta's gene therapy approach. Vertex and CRISPR haven't actually edited any DNA in human muscle cells yet, but Sarepta's approach to treating DMD has already exceeded expectations ...