CureDuchenne is pleased to share the following updates from Pfizer pertaining to the on-going multicenter Phase II clinical trial of the investigational compound, domagrozumab (PF-06252616) in boys with Duchenne muscular […]
Guest blog by Paul Miller Our son Hawken was diagnosed with Duchenne in the fall of 2002. Debra and I rolled up our sleeves and began a journey we had […]
As a community, we need to continue our efforts to find a cure for every single person impacted by Duchenne. That means funding research that treats all aspects of the […]
Yesterday the House of Representatives passed the draft of the 21st Century Cures Act. It is slated for a vote in the Senate early next week. The […]
Thanksgiving is more than a chance to appreciate what we have. It’s also about taking the time to appreciate the people who help us in our journey, those who share […]
The final decisions could be made today on the 21st Century Cures Act and the OPEN ACT – a provision that could be transformational for rare disease patients. CureDuchenne […]
Understanding a patient’s motivation for treatment is the key for a physical therapist to empower that patient for the best outcome. For a patient with Duchenne muscular dystrophy, a […]
Thank you to Marathon Pharmaceuticals for providing updated information about deflazacort access and regulatory status during a webinar this week. The webinar addressed Marathon’s Expanded Access Program for deflazacort and […]
As a parent of a son with Duchenne I have been frustrated that the data presented to the FDA does not match the patient experience. I personally know so […]
