Ana Pena, PhD,  —

Lithium chloride — once used as table salt — improved muscle size and strength in mouse models of limb-girdle muscular dystrophy D1 (LGMD1) by blocking a protein that is overactive in some people with this disease. Inhibiting this protein may be a way of correcting muscle weakness in these…

Researchers at the University of California in Los Angeles (UCLA) have been awarded a $175,000 grant from Parent Project Muscular Dystrophy (PPMD) to make CRISPR/Cas9 as safe and effective as possible as a gene therapy tool to treat Duchenne muscular dystrophy (DMD). According to the researchers, their planned CRISPR/Cas9 gene…

The Institute for Clinical and Economic Review (ICER) issued a draft report raising serious concerns about the cost-effectiveness of Sarepta‘s exon-skipping therapies Exondys 51 (eteplirsen), and the investigational golodirsen, as well as the corticosteroid Emflaza (deflazacort; by PTC Therapeutics), for treating…

Fulcrum Therapeutics announced it has acquired global rights to develop and potentially market losmapimod, an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD), and that it plans to open a Phase 2 clinical trial testing the compound’s safety and efficacy this year. The compound was initially developed by GlaxoSmithKline (GSK) for…

A single dose of gene therapy based on CRISPR-Cas9 may successfully treat Duchenne muscular dystrophy (DMD) in the long term or even permanently, a preclinical study in mice shows. The findings shed light on safety factors that may affect the long-term efficacy of this gene therapy, and provide new evidence…

Cugamycin, a small molecule compound targeting the disease-causing RNA repeats in people with myotonic dystrophy type 1 (DM1), showed promise in improving muscle defects — without evident side effects — in an early study using mouse and cell models, researchers report.

Restoring a functional dystrophin gene using the genome editing tool  CRISPR-Cas9 corrected a common mutation behind Duchenne muscular dystrophy (DMD), exon 44 deletion, in mice and human cells, a study shows. The same…

Sarepta Therapeutics has entered a long-term, strategic partnership with Aldevron to advance its gene therapy program for Duchenne mulcular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). Aldevron, which produces biological products for the biotech industry, will be responsible for supplying…

WVE-210201, an exon 51 skipping therapy, has shown positive safety and tolerability results in a Phase 1 clinical trial in boys with Duchenne muscular dystrophy (DMD), Wave Life Sciences announced. According to Wave Life Sciences, the positive safety data supports moving the therapy into a Phase 2/3 trial, planned to start…

Muscle biopsies can benefit the diagnosis and treatment of neuromuscular diseases in children, particularly those with muscular dystrophy or inflammatory muscle disease, a study suggests. In the study, “Does muscle biopsy change the treatment of…