Patricia Inácio, PhD,  science writer—

Researchers have found that certain cognitive functions, specifically those requiring sequential processing of auditory and visual information, do not improve with age in patients with Duchenne muscular dystrophy (DMD). The study, “Profile of cognitive function in adults with duchenne muscular dystrophy,” was published in the journal Brain and…

Translarna (ataluren) is a promising drug for the treatment of Duchenne muscular dystrophy (DMD) and cystic fibrosis. A team of scientists at University of Massachusetts Medical School and the University of Alabama at Birmingham has provided additional knowledge into Translarna’s mechanism of action, advancing our understanding of how the drug works to…

Researchers have found that a specific protein, CD82, is a marker for muscle stem cells and its expression is reduced in muscular dystrophy patients, suggesting a potential role for this previously unknown protein in the disease. The study, “CD82 Is a Marker for Prospective Isolation of Human Muscle Satellite Cells…

An international team of researchers has reviewed the latest treatments targeting non-muscle cells in the heart responsible for cardiac scarring. The information is important for developing strategies to treat diseases that are often viewed as hopeless, from common ailments like advanced heart failure to rare but deadly ones like muscular dystrophy. The study from…

Catabasis Pharmaceuticals, in a joint initiative with Parent Project Muscular Dystrophy (PPMD), recently hosted the webinar “MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy.” The clinical-stage biopharma company, together with PPMD, a nonprofit organization founded in 1994 by parents of children with Duchenne and…

University of Chicago researchers developed a DNA nanosensor that measures the physiological concentration of chloride with a high degree of accuracy, a discovery with critical implications for diseases such as muscular dystrophies. Until now, there was no effective way to measure intracellular stores of chloride. The newly developed sensor, called…

Deflazacort, a glucocorticoid drug, was shown to improve muscle strength in boys with Duchenne muscular dystrophy (DMD) in a clinical trial performed by Marathon Pharmaceuticals. Trial results were reported in a presentation, “Effect of DEFLAZACORT and PREDNISONE versus placebo on muscle strength in boys with duchenne muscular dystrophy who have lost ambulation: results…

FibroGen, a biotechnology company with expertise in connective tissue growth factor (CTGF) and hypoxia-inducible factor (HIF) biology, recently announced enrolling the first two patients in its new, open-label, multicenter Phase 2 clinical trial to investigate the FG-3019 compound in Duchenne muscular dystrophy (DMD) patients.  The trial is currently recruiting participants and further…

In a new study entitled “Jagged 1 Rescues the Duchenne Muscular Dystrophy Phenotype,” a team of researchers identified a single gene – Jagged1 – as the potential critical player capable of “rescuing” Duchenne muscular dystrophy phenotype in dogs that exhibit a complete absence of dystrophin. The study was published…

Debiopharm International SA, in partnership with Solid Biosciences, recently announced the initiation of pre-clinical animal studies of Alisporivir in the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a severe form of muscular dystrophy caused by a mutation in the Dystrophin gene, located in the X chromosome, which results in the absence or abnormal production of…