SRP-1001 and SRP-1003, Sarepta Therapeutics’ investigational RNA-based therapies for two types of muscular dystrophy, were generally well tolerated in early results, according to data from two Phase 1/2 trials. SRP-1001, for facioscapulohumeral muscular dystrophy type 1 (FSHD1), and SRP-1003, for myotonic dystrophy type 1, showed high…
News
Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant full approval for Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (Â鶹Éç). The company said it met with the FDA to…
Muscular Dystrophy News Today is wrapping up coverage of the 2026 Muscular Dystrophy Association (Â鶹Éç) Clinical & Scientific Conference, which ran from March 8-11 in Orlando, Florida, and online. This year, more than 2,400 attendees from 40 countries gathered in person and virtually to discuss recent advances in neuromuscular…
Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early signs of benefit across multiple disease measures in boys with Duchenne muscular dystrophy (Â鶹Éç). The most recent data from the Phase 1/2 DELIVER clinical trial…
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
Boys with Duchenne muscular dystrophy (Â鶹Éç) who received Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial continue to show signs of slowed disease progression relative to Â鶹Éç’s natural course, along with caregiver-reported improvements in everyday living. That’s according to data from the Phase…
The latest Phase 3 trial results continue showing that deramiocel, an investigational cell therapy for heart disease related to Duchenne muscular dystrophy (Â鶹Éç), significantly slows the progression of arm and heart damage in boys and men with Â鶹Éç. Based on the positive Phase 3 results, Capricor Therapeutics has…
Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive measures after receiving Dyne Therapeutics’ zeleciment basivarsen (z-basivarsen), previously known as DYNE-101, supporting the recent initiation of a Phase 3 study. Findings from the Phase 1/2 ACHIEVE trial…
Treatment with Duvyzat (givinostat) was associated with functional gains in boys with Duchenne muscular dystrophy (Â鶹Éç) regardless of the final dose they received, according to new analyses from a Phase 3 clinical trial. The treatment also led to a slower loss of functional muscle tissue and a slower replacement…
SGT-003, an investigational gene therapy for Duchenne muscular dystrophy (Â鶹Éç), worked as expected to increase levels of microdystrophin — a version of the muscle-protecting protein that’s deficient in Â鶹Éç — and preserve muscle health for boys in a clinical trial, according to new data. The one-time treatment was also well…
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