News

Oral treatment with EDG-5506, now known as sevasemten, continued to stabilize muscle function after two years in men with Becker muscular dystrophy (BMD) taking part in a Phase 1 trial. That’s in contrast to the progressive deterioration of motor function seen during the natural course of BMD in the absence…

Treatment with an antioxidant supplement led to improvements in muscle strength and quality of life for people with facioscapulohumeral muscular dystrophy (FSHD) in a small clinical trial. The use of such supplements was particularly seen to improve the muscle quality of patients’ quadriceps — a group of muscles found…

Diagnostic testing of individuals in India suspected of having limb-girdle muscular dystrophy (LGMD) revealed two novel mutations in the calpain-3 gene, known as CAPN3, that were found to cause the common subtype R1, known as LGMDR1. These findings were detailed in a new study reporting the results of testing…

Up to eight years of Exondys 51 treatment (eteplirsen or AVI-4658) extended survival in Duchenne muscular dystrophy (DMD) patients over a wide range of ages, a long-term study concluded. Patients treated for longer periods had the lowest risk of death. The study, “Survival among patients…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease status to SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) developed by Solid Biosciences. The designation is given to therapies with the potential to prevent or treat rare diseases that primarily affect children and…

ITF Therapeutics — which will be responsible for marketing Duvyzat (givinostat), Italfarmaco’s newly approved Duchenne muscular dystrophy (DMD) therapy, in the U.S — expects the oral medication to be available to eligible adults and children by this fall. For ITF, the U.S.-based rare disease division of Italfarmaco,…

Regulators in France and Italy have given Atamyo Therapeutics the green light to launch a clinical trial testing ATA-200, an investigational gene therapy for children with a type of limb-girdle muscular dystrophy called LGMD2C or LGMDR5. The multicenter, dose-escalation Phase 1b study (NCT05973630) — which will assess…

In myotonic dystrophy type 2 (DM2), muscle cells have dysfunctional mitochondria, the cell’s so-called powerhouse that’s key to energy generation. The results imply that treatments to boost mitochondrial function could be beneficial in DM2, the researchers said in “Multi-level profiling unravels mitochondrial dysfunction in myotonic dystrophy…

The U.S. Food and Drug Administration (FDA) has approved Italfarmaco’s givinostat, which will now be marketed as Duvyzat, as an oral suspension to treat people with Duchenne muscular dystrophy (DMD) ages 6 and older. Duvyzat becomes the first nonsteroidal therapy approved in the U.S. for patients with all…

Treatment with givinostat — an oral therapy currently up for approval in the U.S. and Europe — significantly slowed motor function loss among boys with Duchenne muscular dystrophy (DMD) in the Phase 3 clinical trial EPIDYS, according to the full study results. Those results were detailed in a…