News

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…

Long-term treatment with AOC 1001 continues to be safe and may reverse the progression of myotonic dystrophy type 1 (DM1), according to new data. A year of treatment eased myotonia, that is, the inability of muscle to relax after they’ve contracted, and improved muscle strength along with outcomes…

Patients and experts in limb-girdle muscular dystrophy (LGMD), along with drug developers, community leaders, and regulatory agency personnel, came together for the LGMD Scientific Workshop to discuss how to tackle the unmeet therapeutic needs of people with these muscle-wasting diseases. The workshop, held in early February in Maryland,…

A cell-based study revealed the biological mechanism behind the increased tendency for people with myotonic dystrophy type 2 (DM2) to develop autoimmune diseases. Researchers found that the genetic defect that causes DM2, called a repeat expansion, indirectly triggered an abnormal, antiviral immune response in patient cells. “That was our…