News

Most people with myotonic dystrophy type 1 (DM1) develop heart problems, according to a Danish study that followed patients for about 10 years. “Life-long cardiac screening is crucial, and we recommended that repeated follow-up is performed,” its researchers wrote in “Natural history of cardiac involvement in myotonic dystrophy type 1 – Emphasis…

Glucocorticoid use can help maintain breathing ability and arm function in adults with Duchenne muscular dystrophy (DMD), a new study reports. Importantly, the study found that glucocorticoid treatment was shown to be helpful even when only considering the time after patients had lost the ability to walk — which…

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…