DMD gene therapy GNT0004 set to enter Phase 3 trial in Europe, US

GNT0004, an experimental gene therapy for Duchenne muscular dystrophy (DMD), appears to be working as intended in the initial parts of a multiphase clinical trial, with benefits including stable or improved motor function. That’s according to data presented by Genethon, the therapy’s developer, at the ASGCT Breakthroughs in…

Genetics, Motor Milestones May Help Distinguish DMD From BMD

Analyzing the type of mutation in the DMD gene combined with assessing motor milestones may help better distinguish Duchenne muscular dystrophy (DMD) from Becker muscular dystrophy (BMD) early in life, a study found. Combining both approaches had better predictive power than using either alone, and could mean that children will…

DMD Treatment Candidate DYNE-251 Granted FDA Fast Track Status

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Dyne Therapeutics’ DYNE-251 for the treatment of people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping. Fast track status is intended to accelerate a therapy’s development and expedite…

HOPE 2 Study: CAP-1002 Still Improving Arm Function in DMD

The investigational cell therapy CAP-1002 continued to improve arm function in boys and young men with advanced Duchenne muscular dystrophy (DMD), according to the HOPE-2 open-label extension study. These additional upper limb improvements came after HOPE-2 participants were off CAP-1002 for a mean of about one year before…