Alice Melão,  —

Scientists have designed two proteins that stabilized muscle cell membrane support and maintained the structure of muscle fibers in animal models of congenital muscular dystrophy (MD). The finding, from the University of Basel in Switzerland, can not only provide new insights into the underlying mechanisms of muscular dystrophy, but may…

Results of the Phase 2 MoveDMD trial showed that Catabasis Pharmaceuticals’ candidate drug edasalonexent (CAT-1004) can reduce the rate of functional decline in boys ages 4–7 with Duchenne muscular dystrophy (DMD). The recent findings will…

Researchers at Saint Louis University found that a nuclear receptor called REV-ERB is an important regulator of different steps in the process of muscle cell regeneration. According to the study “Rev-Erb co-regulates muscle regeneration via tethered interaction with the NF-Y cistrome,” which was featured in the journal Molecular…

Simultaneously inhibiting three proteins can increase muscle mass, a finding that could help those with diseases such as muscular dystrophy and cancer that cause muscle deterioration, an Australian study reports. The study, “Specific targeting of TGF-β family ligands demonstrates distinct roles in the regulation of muscle mass in health and…

A former investigational drug initially designed to target cancer was found to have therapeutic potential for Duchenne muscular dystrophy (DMD), according to an article published in the scientific journal Molecular Therapy. The study, “SU9516 Increases α7β1 Integrin and Ameliorates Disease Progression in the mdx Mouse Model of Duchenne…

Genetic screening for the presence of mutations that characterize Duchenne and Becker muscular dystrophies (DMD and BMD) in women before conception can help determine the risk their offspring will develop either disease, according to a Polish study published in the Journal of Obstetrics Gynaecology Research. The study, “Prenatal diagnosis of Duchenne…

Positive results from the HOPE-Duchenne clinical trial support further clinical development of Capricor Therapeutics’ therapy candidate CAP-1002 for treatment of cardiac anomalies caused by Duchenne Muscular Dystrophy (DMD). The company expects to start enrolling patients for a placebo-controlled clinical trial of intravenous CAP-1002 in DMD in the second…

Acceleron Pharma said the first patient has been dosed in a Phase 2 clinical trial of ACE-083, its proprietary lead candidate drug ACE-083 to treat facioscapulohumeral muscular dystrophy (FSHD) — a genetic disease affecting 19,000 Americans for which no approved therapy yet exists. “ACE-083 has generated a strong data set in…

Researchers from the University of Texas Southwestern Medical Center in Dallas have corrected Duchenne muscular dystrophy (DMD) mutations in human cells and experimental animal models using a new genome editing method called CRISPR-Cpf1. These new findings offer hope in permanently eliminating genetic mutations and rescue abnormalities linked to DMD. The study, “…

Researchers investigated swallowing dysfunctions in Becker muscular dystrophy (BMD) patients and found they have swallowing problems similar to those observed in patients with Duchenne muscular dystrophy (DMD). These observations are important not only for a better understanding of BMD progression, but also to improve the standard care of these patients. The…