A Phase 2 trial of ATL1102 met its primary goal of demonstrating favorable safety and tolerability in boys with Duchenne muscular dystrophy (DMD), final study results show. ATL1102, developed by Antisense Therapeutics, was also effective at lowering the number of immune cells targeted by its mechanism of action, improving upper…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Epirium Bio’s EB 002 ((+)-epicatechin) for the treatment of Duchenne and Becker muscular dystrophies. The designation supports the development of experimental treatments for rare diseases (those that affect fewer than 200,000 people in the…
Capricor Therapeutics will host a conference call and webcast with a cardiac expert to discuss heart complications associated with Duchenne muscular dystrophy (DMD) and will also provide an update on the development of CAP-1002, its lead cell therapy candidate for people with DMD. The event, at 1:30…
Fulcrum Therapeutics announced that its Phase 2b trial assessing the safety and efficacy of losmapimod, an investigational oral treatment for facioscapulohumeral muscular dystrophy (FSHD), is fully enrolled. The trial (NCT04003974), called ReDUX4, started recruiting patients at clinical sites in the U.S., Canada, and Europe last year. It is…
Treatment with chloroquine increased the levels and restored the activity of Muscleblind-like RNA-binding proteins, improving muscle function and strength in animal models of myotonic dystrophy type 1, a study has found. The study, “Increased Muscleblind levels by chloroquine treatment improve myotonic dystrophy type 1 phenotypes in in…
The gene-editing tool CRISPR/Cas9 was used to correct defects in the DMD gene and restore dystrophin protein production,  lengthening the lives of pigs in a model of Duchenne muscular dystrophy (DMD) and altering heart cells from a patient to make them less prone to irregular beats, researchers report. The scientists…
GeneDx has chosen Bionano Genomics’ technology, called Saphyr System, to aid in developing tests to detect mutations in genes that are associated with different forms of muscular dystrophy and other disorders. Saphyr is a whole-genome imaging technology that uses advanced optics combined with mathematical…
Note: This story was updated Dec. 17, 2020, to note that preclinical studies testing this form of gene therapy were conducted in animals that naturally develop DMD, not genetically engineered animals. Genethon and Sarepta Therapeutics have extended their original agreement to develop GNT0004, a potential …
Solid Biosciences announced it will reduce its workforce by approximately one third to cut expenses and focus on advancing the development of SGT-001, its gene therapy candidate for the treatment of Duchenne muscular dystrophy (DMD). SGT-001 uses a viral vector to deliver a…
Astellas Pharma recently agreed to acquire Audentes Therapeutics, a move it expects will result in faster development of potentially best-in-class therapies for rare neuromuscular diseases, including muscular dystrophy (MD). Audentes’ vectorized exon-skipping technology — which uses a modified adeno-associated virus (AAV) vector to allow cells to…
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