Margarida Maia, PhD, science writer —

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…

The European Medicines Agency (EMA) has started reviewing an application to market givinostat for Duchenne muscular dystrophy (DMD). “Now that our market authorization application is under review, we are looking forward to working with the regulatory authorities to bring this treatment option to patients,” Paolo Bettica, MD, PhD,…

Pamrevlumab, an antibody in FibroGen’s pipeline, did not improve motor skills in boys with Duchenne muscular dystrophy (DMD), ages 6 to 11, who could walk, according to top-line data from a Phase 3 study. The LELANTOS-2 (NCT04632940) trial tested against a placebo pamrevlumab’s safety and how…

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease status to SAT-3153, Satellos Bioscience’s first-in-class therapy candidate to rebuild muscle tissue in Duchenne muscular dystrophy (DMD). “Receiving orphan drug designation and rare pediatric disease designations are important milestones in advancing our…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy status to NS-089/NCNP-02, NS Pharma’s candidate for Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping, the company announced. The designation is intended to speed up the development and review of therapies for serious or life-threatening…