Capricor Therapeutics will soon meet with the U.S. Food and Drug Administration (FDA) to discuss plans for an application seeking approval of the company’s CAP-1002 cell therapy, now named deramiocel, as a treatment for Duchenne muscular dystrophy (DMD). The FDA has scheduled the pre-biologics license application (BLA)…
News
The Parent Project Muscular Dystrophy (PPMD) 30th annual conference kicks off today, offering families affected by Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) the chance to “celebrate three decades of progress, community, and resilience.” The PPMD conference, which runs through Saturday, is taking place…
A Phase 3 trial testing an experimental therapy for limb-girdle muscular dystrophy type 2i (LGMD2i) has surpassed patient enrollment for a planned interim analysis, the therapy’s developer BridgeBio Pharma has announced. The Phase 3 FORTIFY trial (NCT05775848) is evaluating the long-term safety and efficacy of BBP-418 in…
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…
Recent Posts
- Finding time for some quiet thoughts amid the travel and doctor visits
- MDA 2026: SGT-003 gene therapy shows early promise for DMD in trial
- MDA 2026: Early trial of PGN-EDODM1 shows biomarker activity in DM1
- As my FSHD progression worsens, I’m battling feelings of shame
- MDA 2026: Sevasemten halts functional decline in Becker trial