News

Sarepta Therapeutics receives Complete Response Letter from the US Food and Drug Administration for golodirsen New Drug Application Sarepta Therapeutics, Inc announced it had received a Complete [...]

Thanks to Bio Space, IOS Press, Science Daily for the content of this blog. Duchenne muscular dystrophy is characterized by progressive muscle degeneration. In DMD patients, the pulmonary [...]

PTC Provides Update on Translarna™ (ataluren) Application for Label Expansion June 28, 2019 > Original press release < PTC Therapeutics, Inc. announced that the Committee for Medicinal [...]

La Force DMD is seeking your participation in a research survey for parents or guardians of sons with Duchenne muscular dystrophy and adult men (18+ years) with Duchenne. This is an international [...]

Clinical Trials Simplified and La Force DMD join forces to accelerate the development of new therapies for DMD During a conference about rare diseases, Marie-Catherine Du Berger, president of La [...]

– 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, [...]

Audentes Therapeutics, Inc., a leading Adeno-associated virus (AAV)* based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular [...]

Catabasis is enrolling boys ages 4 to 7 (up to 8th birthday), any mutation type, who have not been on steroids for at least the past six months.   What is Edasalonexent? Edasalonexent [...]

Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, announced positive results from its interim analysis of muscle biopsy endpoints comparing casimersen [...]

Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53 Press release here: Sarepta.com   [...]