News

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…

DYNE-101 and DYNE-251, Dyne Therapeutics’ investigational therapies for forms of muscular dystrophy, continue to work as intended and may lead to functional improvements for patients, according to new clinical trial data. These findings come from ongoing Phase 1/2 studies, which may still be recruiting. DELIVER (NCT05524883), launched…

Findings from a Phase 2 trial testing losmapimod in adults with facioscapulohumeral muscular dystrophy (FSHD) have offered new insights into the best outcome measures for future clinical trials, according to final published data from the clinical study, which wrapped up in 2021. The  Fulcrum Therapeutics treatment, as…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to delpacibart etedesiran (del-desiran, previously called AOC 1001), an investigational treatment for myotonic dystrophy type 1 (DM1) that’s about to enter Phase 3 clinical testing. The FDA gives the designation to experimental therapies that have the…