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WITH GRATITUDE THIS THANKSGIVING
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 […]
“My son Aiden was diagnosed with Duchenne Muscular Dystrophy on June 2, 2015. He was 3 at the time. That date will forever be etched in my mind…”
My heart was beating so fast, anxiety creeping into my body. It felt like everything stopped. I can’t remember her exact words, but I distinctly remember the fear that took […]
Help Support the 21st Century Cures Act and the OPEN ACT
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 […]
Spotlight on Sandi Preston, PT, CureDuchenne Cares Certified Physical Therapist
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 […]
CHMP Recommends Renewal of Translarna’s™ Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy Based on Continued Positive Benefit-Risk Assessment
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.
Capricor Therapeutics Announces Plans to Expand Clinical Development Program in Duchenne Muscular Dystrophy to Evaluate Peripheral and Respiratory Muscle Function
Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other serious medical conditions, today announced that it intends to expand its CAP-1002 clinical development program in Duchenne muscular dystrophy (DMD) to encompass the skeletal muscle aspects of the disease, in addition to the cardiac complications.
“As part of our journey, our family has partnered with CureDuchenne, a national non-profit that is leading the effort to accelerate the drug approval process on potential life-saving therapies…”
Our Journey Deaing with Duchenne Our son Joshua was born in 2007. Joshua, our oldest son, is an amazing and fun-loving boy. He was diagnosed with Duchenne Muscular Dystrophy in 2013. […]
Marathon Webinar: Update on Deflazacort Access and Regulatory Status
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 […]
RASRx
Approach: Anti-fibrotic
Funding Impact: Accelerated identification of lead compound and de-risked pre-clinical program