Today, we are very pleased to announce that Pfizer has acquired Bamboo Therapeutics, a gene therapy company we invested in earlier this year through CureDuchenne Ventures. The deal is valued […]
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 […]
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 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 […]
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 […]
From Paul Miller Another Father’s Day is here and I wish you all a meaningful day filled with memories that will last a lifetime. As I think about the most […]
BioMarin is providing a further update to the Duchenne Community in light of recent announcements of withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development […]
Gene therapy proposes a promising approach in treating Duchenne, placing corrected genes into cells that have missing or damaged genes. Although gene therapy is still relatively early, CureDuchenne is […]
We are sharing the following update from BioMarin. We would like to update the patient community in light of the recent announcement regarding the EMA marketing application. BioMarin is […]
