Lila Levinson, PhD,  Science Writer—

An experimental gene editing therapy for Duchenne muscular dystrophy (DMD) being developed by Precision Biosciences has received orphan drug status from the U.S. Food and Drug Administration (FDA). The designation focuses on treatments for rare diseases like DMD. Its benefits include tax credits and fee exemptions, along with…

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…

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…

The European Comission has conditionally approved Duvyzat (givinostat) to be taken alongside corticosteroids for treating people who are 6 and older with Duchenne muscular dystrophy (DMD). “This milestone means that a broad range of patients with DMD have access to a new treatment,” Francesco De Santis, president of…

Satellos Bioscience’s SAT-3247 was safe, well-tolerated, and showed initial signs of increasing muscle strength in five adults with Duchenne muscular dystrophy (DMD), the company said. “We believe the findings from this Phase 1b study support our plan to advance SAT-3247 into a placebo-controlled Phase 2 trial,” Satellos CEO…

The Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency known as CHMP for short, has recommended the approval of Duvyzat (givinostat) as a treatment for people with Duchenne muscular dystrophy (DMD), ages 6 and older, who are able to walk. The European…