Santhera Pharmaceuticals has submitted a marketing authorization request to the European Medicines Agency (EMA) for Puldysa (idebenone) as a treatment…
Articles by Patricia Inácio, PhD
New nine-month data on four boys with Duchenne muscular dystrophy (DMD) enrolled in Study-101 testing Sarepta Therapeutics‘ micro-dystrophin gene therapy…
Santhera Pharmaceuticals intends to file for a conditional marketing authorization with the European Medicines Agency (EMA) for idebenone…
Young boys with Duchenne muscular dystrophy (DMD) have a higher rate of bone fractures than healthy children, a retrospective study…
The Institute for Clinical and Economic Review (ICER) has set up a draft document to review evidence of the effectiveness…
The Hippo signaling pathway — a key regulator of tissue development, and cell growth and death — does not work…
Sarepta Therapeutics‘ microdystrophin gene therapy enhanced dystrophin protein expression in the muscles of four boys with Duchenne muscular…
The U.S. Food and Drug Administration has lifted the clinical hold on a Phase 1/2 trial evaluating Sarepta Therapeutics‘ micro-dystrophin …
Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy…
PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision and Empowerment…