Alice Melão,  —

An advisory committee of the European Medicines Agency (EMA) has issued another negative recommendation after re-examining a request to extend Raxone (idebenone) for use in treating Duchenne muscular dystrophy (DMD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) issued its first negative opinion of the request in…

A dysfunctional cerebellum leads to implicit learning deficits in children with Duchenne muscular dystrophy (DMD), Italian researchers have suggested. Their study, “Implicit learning deficit in children with Duchenne muscular dystrophy: Evidence for a cerebellar cognitive impairment?” appeared in the journal PLoS ONE. DMD is an…

Clinical data from a Phase 3 trial revealed that Kyndrisa (drisapersen) could not significantly improve the walking ability of boys with Duchenne muscular dystrophy (DMD) due to mutations amenable with exon 51 skipping. Still, additional analysis of the DEMAND III trial (NCT01254019) showed this therapy may still…

Apple Tree Partners has invested $40 million in Stoke Therapeutics to come up with gene-expression treatments for muscular dystrophy and other genetic disorders. Stoke aims to develop antisense oligonucleotides to promote targeted augmentation of nuclear gene output (TANGO). The goal is to reverse the features of rare diseases…

Treatment with Exondys 51 (eteplirsen) can prevent lung function decline in patients with Duchenne muscular dystrophy (DMD), according to the results of an analysis of clinical trial data. The findings were reported in a study titled “Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison…

UCLA researchers have developed a new method to efficiently produce and transplant functional skeletal muscle cells from human pluripotent stem cells (hPSCs). This may offer new therapeutic opportunities for patients who have muscle diseases such as Duchenne muscular dystrophy (DMD). The findings were reported in an article titled “…

Benitec Biopharma has submitted an application for orphan drug designation with the U.S. Food and Drug Administration (FDA) for its lead gene therapy candidate, BB-301, for the treatment of oculopharyngeal muscular dystrophy (OPMD). If granted, the orphan drug status will guarantee seven-year market exclusivity upon treatment approval, but…

The absence of a signaling protein leads to immature muscle cells being unable to fuse together to promote muscle tissue growth, a study shows. University of Louisville researchers said lack of the MyD88 signaling protein prevented immature muscle cells known as myoblasts from fusing. When the protein’s levels are normal,…

The researchers who developed the CRISPR DNA editing method that has taken medicine by storm have come up with a tweak to the technique that has implications for Duchenne muscular dystrophy. They can now edit single units of RNA code without modifying genetic content. RNA are building blocks of life that…

Infection by the influenza virus, commonly known as the flu, was found to contribute to muscle degeneration in a zebrafish model of Duchenne muscular dystrophy (DMD). Scientists often use zebrafish in research due to their genetic structure, which is similar to that of humans. The findings were reported in the…