Patricia Inácio, PhD, science writer —

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…

In a new study entitled “Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells” a team of scientists developed an induced pluripotent stem cell model to study Duchenne muscular dystrophy and discover new therapeutic drugs. The study was published in the journal…

In a new study entitled “Differential Isoform Expression and Selective Muscle Involvement in Muscular Dystrophies”researchers performed a microarray analysis on human muscles to identify the molecular cause underlying the selectivity of muscles affected in muscular dystrophies. Their findings suggest that overexpression of mutated genes in specific muscles are…

In a new study entitled “Genome-wide Mechanosensitive MicroRNA (MechanomiR) Screen Uncovers Dysregulation of their Regulatory Networks in the mdm Mouse Model of Muscular Dystrophy” researchers performed a genome-wide study to identify whether mechanosensitive microRNAs are deregulated and contribute to muscular dystrophies pathogenesis. The study was published in…