News

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…

Agamree (vamorolone) is now available for Duchenne muscular dystrophy (DMD) patients, ages 2 and older, in the U.S., where it will be dispensed through a specialty pharmacy network. The U.S. Food and Drug Administration (FDA) approved the next-generation steroid, expected to be more tolerable than standard…

The investigational cell therapy CAP-1002 has continued to slow declines in upper limb and heart function among boys and young men with Duchenne muscular dystrophy (DMD), according to two-year results from the HOPE-2 open label extension (OLE) study. The findings were presented at the recent 2024 Muscular Dystrophy…

DYNE-251 and DYNE-101, Dyne Therapeutics’ investigational therapies for forms of muscular dystrophy, appear safe and are showing signs of preliminary efficacy in Phase 1/2 clinical trials. These proof-of-concept data, announced by Dyne in January, were discussed in two oral presentations at the recent 2024 Muscular Dystrophy…

Among ambulatory boys with Duchenne muscular dystrophy (DMD), a one-time treatment with investigational gene therapy fordadistrogene movaparvovec has helped preserve functions and increase muscle volume for three years, especially in the youngest patients. That’s according to updated analyses from a Phase 1b trial (NCT03362502), in which the…