Translarna™ (ataluren) is the First Therapy Approved in Brazil for DMD
– Ambulatory Duchenne patients who are five years and older with a nonsense mutation can now access a treatment that targets the underlying cause of DMD – PTC Therapeutics, [...]
The brother movie: Stuck between a rich inner life and daily struggles with Duchenne
The brother movie is the story of the Karouani family in France and their daily life with Kaïs, their son, who has Duchenne muscular dystrophy (DMD). Duchenne is a degenerative muscle disease. In [...]
FibroGen receives orphan drug designation for Pamrevlumab
FibroGen Receives Orphan Drug Designation from the U.S. FDA For Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy FibroGen, Inc. a leading biopharmaceutical company discovering [...]
New gene therapy for Duchenne muscular dystrophy
Audentes Therapeutics, Inc., a leading Adeno-associated virus (AAV)* based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular [...]
