News

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…

Del-desiran for DM1 wins FDA’s breakthrough therapy designation

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to delpacibart etedesiran (del-desiran, previously called AOC 1001), an investigational treatment for myotonic dystrophy type 1 (DM1) that’s about to enter Phase 3 clinical testing. The FDA gives the designation to experimental therapies that have the…

AMO Pharma to start AMO-02 Phase 3 trial in adult-onset DM1

AMO Pharma has announced it will conduct a Phase 3 trial of AMO-02 (tideglusib), its investigational oral therapy for adult-onset myotonic dystrophy type 1 (DM1). The decision follows a recent meeting with the U.S. Food and Drug Administration (FDA) to review data from the Phase 2/3…

Baby Duchenne research network receives $250K from PPMD

A $250,000 award from the Parent Project Muscular Dystrophy (PPMD) will support the establishment of Baby Duchenne, a collaborative clinical research network for babies with Duchenne muscular dystrophy (DMD) diagnosed via newborn screening (NBS) programs in New York state. Under the direction of Bo Hoon Lee, MD, from…