Summit Therapeutics announced that it has received a Rare Pediatric Disease designation from the U.S. Food and Drug Administration…
Articles by Charles Moore
The U.S. Food and Drug Administration has granted accelerated approval to Exondys 51 (eteplirsen), by Sarepta Therapeutics, making it…
Australia’s drug regulatory agency, the Therapeutic Goods Administration (TGA), has granted Santhera Pharmaceuticals’ lead product Raxone (idebenone)…
PTC Therapeutics marked Sept. 7 — World Duchenne Awareness Day — by announcing the four winner of the company’s 2016 global…
CureDuchenne, the Duchenne muscular dystrophy nonprofit group engaged in disease advocacy and research funding, announced on Sept. 7 —…
Summit Therapeutics reports that a new formulation of ezutromid (called F6), its Duchenne muscular dystrophy (DMD) treatment candidate, achieved a…
PTC Therapeutics has provided global regulatory updates on its lead product candidate Translarna (ataluren) for treating nonsense mutation Duchenne muscular…
Two British physiotherapists invited to speak at a July 20 event at the Houses of Parliament in London by …
Santhera Pharmaceuticals reports that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for…
Northbrook, Illinois-based Marathon Pharmaceuticals reports it has submitted a New Drug Application (NDA) to the U.S. Food and Drug…