Pharmaceuticals, Biotechnology and Life Sciences
Catabasis Pharmaceuticals has enrolled more than expected patients for its third phase PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD), for which it expects the top-line results in the fourth quarter of 2020.
cTAP has published a comprehensive analysis of the North Star Ambulatory Assessment (NSAA), a newly adopted primary outcome measure in clinical trials of patients with Duchenne Muscular Dystrophy.
Pfizer will present initial Phase 1b clinical data on PF-06939926, an investigational gene therapy to potentially treat Duchenne muscular dystrophy (DMD) at the 25th Annual Parent Project Muscular Dystrophy (PPMD) Connect Conference in Orlando, FL. These are preliminary data drawn from a small number of participants in an ongoing study.
The U.S. Food and Drug Administration (FDA) has approved Marathon Pharmaceuticals’s Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness.