Patricia Inácio, PhD,  science writer—

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to GBC0905 as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD),its developer, Genea Biocells, announced. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA…

The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency (EMA) — is recommending against Exondys 51 (eteplirsen) as a therapy for Duchenne muscular dystrophy (DMD) patients amenable to skipping exon 51 of the dystrophin gene, the treatment’s developer, Sarepta Therapeutics, has announced. The company…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to investigational therapy Sarconeos for Duchenne muscular dystrophy (DMD), Biophytis, the therapy’s developer, announced. The company also has filed a similar application with the European Medicines Agency (EMA). A decision is is expected…

Exondys 51 (eteplirsen), an RNA-targeted therapy marketed by Sarepta Therapeutics, is the first approved treatment targeting the underlying cause of Duchenne muscular dystrophy (DMD). Sarepta is continuing to refine its RNA-targeted therapies, with a goal of being able to transport more of the treatment…

The investigational therapy edasalonexent was seen to slow disease progression in boys with Duchenne muscular dystrophy, almost one-year results from the MoveDMD trial report. Specifically, edasalonexent — administered orally as 100 mg/kg dose for 48 weeks — resulted in a statistically significant delay in disease progression and signs of…

Magnetic nanotubes with a spear-like tip may enhance the precision and effectiveness of gene therapy delivery, a promising therapeutic strategy for many genetic diseases, including muscular dystrophy, according to UCLA researchers. The study, “Precision-Guided Nanospears for Targeted and High-Throughput Intracellular Gene Delivery,” was published in the journal ACS Nano.

Scientists are increasingly looking at gene therapies as potential treatments for Duchenne muscular dystrophy. But there are a lot of ways to do them, and an expert in the field shed light on the subject in a webinar arranged by Parent Project Muscular Dystrophy (PPMD). Dr. Timothy Cripe discussed the…

Summit Therapeutics has opened enrollment for an additional group in its ongoing PhaseOut DMD trial to include patients with Duchenne muscular dystrophy (DMD) who participated in the previous Phase 1 clinical trials for the investigational therapy ezutromid (SMT C1100) but who, at the time of enrollment, failed to meet the enrollment…

A new interim analysis of the PhaseOut DMD clinical trial shows that ezutromid (SMT C1100) significantly decreased muscle inflammation in patients with Duchenne muscular dystrophy (DMD), Summit Therapeutics announced. Ezutromid is called a utrophin modulator because it is able to trigger utrophin production in people who lack dystrophin,…

PTC Therapeutics is recruiting Duchenne muscular dystrophy (DMD) patients ages 5 and older and with a nonsense mutation for a long-term Phase 3 study to characterize the effects of Translarna (ataluren) on disease progression. The trial (NCT03179631) opened in July 2017 and enrolling patients at eight sites across…