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Thoughts about PTC
The announcement that PTC recently submitted an appeal to elevate the “Refusal to File” of Translarna (also known as ataluren and PTC124) to the next level of FDA management is […]
Santhera Updates on U.S. Regulatory Filing for Raxone ® (idebenone) in Duchenne Muscular Dystrophy (DMD)
Summer Road Trip
Guest blog by Hawken Miller Many of the things I don’t think are possible for me to do really are. Once I get over my initial apprehension and thoughts […]
BioMarin Update for the Duchenne Community: 7th July, 2016
BioMarin is providing a further update to the Duchenne Community following our announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of […]
NHS England Enables Access to Translarna™ ▼ (ataluren) For Patients with Nonsense Mutation Duchenne Muscular Dystrophy
Catabasis Pharmaceuticals Announces the Initiation of an Open- Label Extension for the MoveDMD ® Trial Studying Edasalonexent (CAT -1004) in Duchenne Muscular Dystrophy
An Update for the Duchenne Community
We are sharing the following update from Sarepta Therapeutics. Sarepta Therapeutics is updating the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study […]