News

FDA approves first generic of Emflaza oral suspension for DMD

The U.S. Food and Drug Administration (FDA) has approved the first generic version of Emflaza (deflazacort) oral suspension for the treatment of people with Duchenne muscular dystrophy (DMD). Called deflazacort oral suspension, the medication was approved for DMD patients 5 and older, a slightly older population than the…

Pitolisant found to reduce fatigue, daytime sleepiness in DM1 patients

After nearly three months of treatment with pitolisant, adults with myotonic dystrophy type 1 (DM1) experienced a reduction of excessive daytime sleepiness (EDS) and fatigue. That’s according to data from the Phase 2 trial (NCT04886518), which is testing the safety and efficacy of Harmony Biosciences’ therapy in…

Three years of CAP-1002 still stabilizing arm, heart function

After three years of treatment with Capricor Therapeutics’ experimental cell therapy CAP-1002, people with Duchenne muscular dystrophy (DMD) continue to show benefits in arm and heart function, new data from the HOPE-2 open-label extension (OLE) study shows. Topline data from the Phase 3 HOPE-3 trial (NCT05126758), which…

Advisory board named to shepherd Duchenne MD candidate

Satellos Bioscience has established a clinical advisory board, with experts in drug development and genetic muscle disorders, to help propel SAT-3247, an oral therapy candidate for Duchenne muscular dystrophy (DMD). “The formation of this clinical advisory board marks a major development step for Satellos as we continue our…

Viltepso fails main goal in confirmatory Phase 3 study

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…