Lithium chloride — once used as table salt — improved muscle size and strength in mouse models of limb-girdle…
Articles by Ana Pena, PhD
Researchers at the University of California in Los Angeles (UCLA) have been awarded a $175,000 grant from Parent Project Muscular Dystrophy…
The Institute for Clinical and Economic Review (ICER) issued a draft report raising serious concerns about the cost-effectiveness…
Fulcrum Therapeutics announced it has acquired global rights to develop and potentially market losmapimod, an investigational treatment for facioscapulohumeral…
A single dose of gene therapy based on CRISPR-Cas9 may successfully treat Duchenne muscular dystrophy (DMD) in the long…
Cugamycin, a small molecule compound targeting the disease-causing RNA repeats in people with myotonic dystrophy…
Restoring a functional dystrophin gene using the genome editing tool CRISPR-Cas9…
Sarepta Therapeutics has entered a long-term, strategic partnership with Aldevron to advance its gene therapy program for…
WVE-210201, an exon 51 skipping therapy, has shown positive safety and tolerability results in a Phase 1 clinical trial in boys…
Muscle biopsies can benefit the diagnosis and treatment of neuromuscular diseases in children, particularly those with …