News

The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to delpacibart zotadirsen, known as del-zota, as a treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping. This designation is intended to speed the development and review of therapies for serious conditions when early…

Sarepta Therapeutics is voluntarily halting all shipments of Elevidys (delandistrogene moxeparvovec-rokl) in the U.S. after a request from the U.S. Food and Drug Administration (FDA). The company had initially refused to pause the shipments of its gene therapy for Duchenne muscular dystrophy (DMD) for individuals with DMD who…

The first patient has been dosed as part of a Phase 1b clinical trial of AAV1.SERCA2 (SRD-003), Sardocor‘s one-time gene therapy for cardiomyopathy — a type of heart disease — associated with Duchenne muscular dystrophy (DMD-CM). The multicenter trial, dubbed MUSIC-DMD (NCT06224660), is being conducted in collaboration…

Capricor Therapeutics will resubmit its application for U.S. Food and Drug Administration (FDA) approval of its cell therapy deramiocel to treat Duchenne muscular dystrophy (DMD)-related heart disease, following the agency’s decision to not accept the original filing. According to a company press release, the FDA issued a…

Nearly one-third of people with muscular dystrophy (MD) experience moderate pain, which varies by the type of their MD, a study with more than 1,200 participants suggests. To manage their pain, about a fifth of patients used prescribed pain medications, mostly non-opioid therapies at first, then combined with opioids…

Households incur substantial costs to accommodate people with Duchenne muscular dystrophy (DMD), according to a survey of patient caregivers in the U.S. Costs, such as those for purchasing and/or modifying an accessible vehicle, relocating to or building an accessible home, modifying home entrances, and buying medical equipment, were particularly…

Researchers identified a unique protein signature in the bloodstreams of adults with Becker muscular dystrophy (BMD) and limb-girdle muscular dystrophy (LGMD) that significantly differs from healthy individuals after exercise. “We propose that a subset of circulating proteins may be more indicative of disease progression and/or therapeutic efficacy than…

Ifetroban, Cumberland Pharmaceuticals’ investigational oral therapy, improved heart function and reduced cardiac damage biomarkers in people with Duchenne muscular dystrophy (DMD), according to new trial data. These findings from the Phase 2 FIGHT DMD trial (NCT03340675) were presented at the Parent Project Muscular Dystrophy (PPMD) annual…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Precision Biosciences. The designation provides companies with extra incentives to develop experimental therapies to treat rare diseases that mainly affect…

The U.S. Food and Drug Administration (FDA) is due to convene an advisory committee meeting next month to review the data on deramiocel, an experimental cell therapy up for approval to treat heart disease in people with Duchenne muscular dystrophy (DMD). The FDA has informed Capricor Therapeutics…