News

The U.S. Food and Drug Administration (FDA) has expanded its approval of Elevidys (delandistrogene moxeparvovec-rokl), authorizing the one-time gene therapy for individuals with Duchenne muscular dystrophy (DMD) ages 4 and older regardless of their ability to walk. Previously, the treatment had been approved for DMD children ages 4…

AOC 1020, an investigational treatment by Avidity Biosciences that’s now called delpacibart braxlosiran, or del-brax, helped adults with facioscapulohumeral muscular dystrophy (FSHD) grow stronger muscles and extend their arms farther than they could four months earlier. These are early data from FORTITUDE (NCT05747924), a three-part Phase 1/2…

The gene therapy developed by Pfizer called fordadistrogene movaparvovec failed to significantly improve motor function in boys with Duchenne muscular dystrophy (DMD) who are able to walk and taking part in a Phase 3 study. The company says it will evaluate the next steps for the fordadistrogene…

The U.S. Food and Drug Administration (FDA) has approved the first generic version of Emflaza (deflazacort) oral suspension for the treatment of people with Duchenne muscular dystrophy (DMD). Called deflazacort oral suspension, the medication was approved for DMD patients 5 and older, a slightly older population than the…

After nearly three months of treatment with pitolisant, adults with myotonic dystrophy type 1 (DM1) experienced a reduction of excessive daytime sleepiness (EDS) and fatigue. That’s according to data from the Phase 2 trial (NCT04886518), which is testing the safety and efficacy of Harmony Biosciences’ therapy in…

After three years of treatment with Capricor Therapeutics’ experimental cell therapy CAP-1002, people with Duchenne muscular dystrophy (DMD) continue to show benefits in arm and heart function, new data from the HOPE-2 open-label extension (OLE) study shows. Topline data from the Phase 3 HOPE-3 trial (NCT05126758), which…

Satellos Bioscience has established a clinical advisory board, with experts in drug development and genetic muscle disorders, to help propel SAT-3247, an oral therapy candidate for Duchenne muscular dystrophy (DMD). “The formation of this clinical advisory board marks a major development step for Satellos as we continue our…

An engineered transfer RNA (tRNA) molecule developed by hC Bioscience could be a fix for the protein that’s lacking in Duchenne muscular dystrophy (DMD), supporting the company’s ongoing efforts to identify a lead molecule that may treat the disease. In a study with a mouse model of DMD,…

After nearly a year of treatment, Viltepso (viltolarsen) was well tolerated and tended to increase how fast boys with Duchenne muscular dystrophy (DMD) could stand from a lying position, but not significantly more than a placebo, according to a preliminary analysis of a Phase 3 study. The main…

Duchenne muscular dystrophy (DMD) can make it harder to engage in social activities, and patients often experience grief and frustration over lost function. Family, however, can be an invaluable source of support to help overcome the challenges of the disease. That’s according to a study, “‘You…