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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 […]
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 […]
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 […]
A Father’s Day Letter to My Fellow Duchenne Dads
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 Update for the Duchenne Community: 17 June 2016
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 […]
CLOSER TO A CURE – BAMBOO THERAPEUTICS AND GENE THERAPY WEBINAR
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 […]
BioMarin Update for the Duchenne Community: 01 June 2016
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 […]
Going Backwards to Go Forwards
I imagine that by now you have heard the news that BioMarin has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) in Europe. Like you, I am heartbroken and […]