Move Duchenne research forward faster with one simple action
Every dollar you give online this holiday season goes toward an important project—the Duchenne Superhighway—that will make the clinical trial process faster and more efficient. And because our Board of Directors has pledged to match your donation dollar for dollar, your gift today makes twice the difference.
Webinar Recording: Update on Natural History Data Study
On November 19th, PPMD hosted a webinar with Craig McDonald, MD to discuss the natural history study he has been a part of and the data that has been collected.
End Duchenne eNews: Time to Unite & Rally
Catch up on the latest research and community updates in this month's End Duchenne eNews. Also meet our Featured Voice for November, 29-year-old Ansel Lurio.
Facing Disappointment from FDA's Eteplirsen Response
The FDA has requested instead that a larger phase III study be conducted before they will consider Sarepta's application for approval. PPMD, which provided travel support to families who participated in the trial, is deeply disappointed in this decision, but hopeful that the FDA will provide clear guidance in the near future on the quantity and quality of data that is required for it to consider an accelerated approval or an application based on phase II data.
Duchenne Policy Forum
PPMD is proud to host a Policy Forum on clinical trials of experiment agents on December 12, 2013 from 8.30AM until 4.30PM in Silver Springs, MD. We are especially pleased that key Duchenne-community stakeholders, including the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER), and review divisions, are committed to the success of this day-long meeting.
Decode Duchenne: A Genetic Testing Program
We are excited to announce that PPMD and Sarepta Therapeutics are joining forces on a new genetic testing program for patients with Duchenne or Becker muscular dystrophy. Through this new program, called Decode Duchenne, PPMD will provide genetic testing at no cost to eligible patients who are unable to access testing due to barriers such as a lack of or insufficient insurance coverage. Sarepta is supporting the PPMD-administered initiative.
End Duchenne eNews: Pushing the FDA to Accelerate Treatments
Catch up on the latest research and community updates in this month's End Duchenne eNews. Also meet our Featured Voice for October, 9-year-old Jake Pritchard.
Pushing the FDA to Accelerate Treatments
PPMD, together with our partners in Washington, FaegreBD, have delivered a new white paper to the FDA, urging them to change their decision-making process when it pertains to rare diseases, especially Duchenne.
Clinical Trial Expectations
PPMD was privileged to share some of the data we have collected in our Clinical Trial Expectations study at the World Muscle Society poster session. Many clinicians and industry and academic scientists came to the poster to learn about our community thoughts and preferences.
WMS Congress: Considering Care
Care related research was popular at this year's 18th International World Muscle Society Congress. Kathi Kinnett, VP of Clinical Care, summarizes some of the highlights.
WMS Congress: Next Generation Exon-Skipping
PPMD is so grateful for you, our community, and our children - our biggest strength. What are you thankful for this year?
November 26, 2013 | Read More
Today, one simple action has twice the impact. Donate now and your gift will be doubled.
Please help us reach our goal of raising $200,000 for an important project that will speed up drug development for Duchenne.
Choose your tax-deductible gift amount: