Margarida Maia, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to MDL-101, an epigenetic editing therapy that Modalis Therapeutics is developing for LAMA2-related congenital muscular dystrophy (LAMA2-CMD) — a genetic condition that’s characterized by muscle wasting evident at birth or shortly thereafter. This FDA status…

AOC 1020, an investigational treatment by Avidity Biosciences that’s now called delpacibart braxlosiran, or del-brax, helped adults with facioscapulohumeral muscular dystrophy (FSHD) grow stronger muscles and extend their arms farther than they could four months earlier. These are early data from FORTITUDE (NCT05747924), a three-part Phase 1/2…

An engineered transfer RNA (tRNA) molecule developed by hC Bioscience could be a fix for the protein that’s lacking in Duchenne muscular dystrophy (DMD), supporting the company’s ongoing efforts to identify a lead molecule that may treat the disease. In a study with a mouse model of DMD,…

After nearly a year of treatment, Viltepso (viltolarsen) was well tolerated and tended to increase how fast boys with Duchenne muscular dystrophy (DMD) could stand from a lying position, but not significantly more than a placebo, according to a preliminary analysis of a Phase 3 study. The main…

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,…

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…

It may take an average of 11 years before people with Duchenne muscular dystrophy (DMD) lose upper body function and the ability to breathe on their own, according to a study by researchers of the HERCULES project, which set out to construct a natural history model of the…

A committee of the European Medicines Agency (EMA) has maintained its initial recommendation not to renew the conditional marketing authorization for Translarna (ataluren), PTC Therapeutics’ medication for Duchenne muscular dystrophy (DMD). The recommendation by the Committee for Medicinal Products for Human Use (CHMP) comes after the company appealed…

Corticosteroids may cause fewer unwanted brain changes in boys with Duchenne muscular dystrophy (DMD) if they are given on and off at 10-day intervals, rather than at daily doses, an imaging study found. “Brain volumetrics [volume measurements] are more profoundly affected in patients treated with a daily corticosteroid…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on PGN-EDODM1, a medicine being developed by PepGen for myotonic dystrophy type 1, or DM1 — a subtype of the most common form of muscular dystrophy. With the hold lifted, PepGen is ready to…