PPMD's 2015 Connect Conference Resources & Recordings
Presentations from PPMD’s 21st Annual Connect Conference in Washington, D.C. are now available for download.
PPMD Awards $70,000 Grant to Talem Technologies as Part of Robotics Initiative
PPMD is proud to award Talem Technologies with a grant for over $70,000 to support a pilot study of their X-Ar exoskeletal arm technology. This grant is a direct result of your donation this spring for our Robotics Initiative. We believe in the X-Ar arm and other technologies that we believe will maintain muscle function and foster independence. Thank you for supporting our robotics program and congratulations to Talem Technologies!
End Duchenne eNews: Celebrating Independence
Catch up on the latest research and community updates in this month's End Duchenne eNews!
PPMD's Duchenne/Becker Transitions Meeting Recap
At PPMD's Duchenne/Becker Transitions Meeting, teens, young adults, parents, and community members gathered to learn about how to ensure that now that people with Duchenne survive into adulthood with greater frequency than ever before — that they also THRIVE in adulthood. If you were unable to join us, you can now access some of the wonderful information that was shared by our presenters online.
Download PPMD's 2015 Connect Conference Booklet
PPMD’s 2015 Connect Conference booklet contained over 40 pages of information on what’s currently going on in Duchenne research & clinical trials. We encourage you to browse the digital version of the booklet if you were unable to attend.
Action ALERT: Urge Congress to Vote YES on 21st Century Cures Act
For over a year, PPMD has been working with Congress on landmark legislation called 21st Century Cures. The bill aims to speed the discovery, development, and delivery of therapies for diseases that have an unmet medical need like Duchenne. This week the House will hold a floor vote on the measure (HR 6). Please take 1 minute to send your House member an Action Alert telling them to vote YES on HR 6, the 21 Century Cures Act.
Revised Duchenne Anesthesia Recommendations
New evidence suggests that people with Duchenne are not at an increased risk of developing malignant hyperthermia, but may continue to be at an increased risk of rhabdomyolysis (serious and sometimes fatal muscle breakdown) when administered inhaled anesthetic gases. In light of this new knowledge, PPMD has joined with experts in muscle disease and anesthesia to clarify our guidelines on the administration of general anesthesia for patients with Duchenne.
FDA Accepts BioMarin's NDA for Drisapersen; Sarepta Completes NDA for Eteplirsen
Big news in exon skipping therapy! BioMarin announced that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen.
Webinar Recording: PF-06252616 (Anti-Myostatin)
On June 3, 2015, Dr. Michael Binks, Vice President for Rare Disease Clinical Research at Pfizer, presented information about the upcoming clinical trial of PF-06252616. The webinar provides an overview of Pfizer’s approach to advancing the science of an anti-myostatin monoclonal antibody as an investigational compound for potential therapeutic use in muscle wasting diseases, such as Duchenne muscular dystrophy.
Live from PPMD's Connect Conference! Urge your Senators to Cosponsor PFIA (S. 1597) from Home!
Yesterday Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) introduced S. 1597, the Patient-Focused Impact Assessment Act, which seeks to drive FDA use of patient focused drug development tools. We need you to reach out to your Senators today and urge that they sign on as a cosponsor of S. 1597.
PPMD's Annual Connect Conference: Streaming Schedule
Can't make it to PPMD's 2015 Annual Connect Conference? We will be live streaming most sessions Thursday, June 18 - Saturday evening, June 20.
PPMD Names Duke Medicine Certified Duchenne Care Center
PPMD is excited to name Duke Children’s the ninth Certified Duchenne Care Center! PPMD recognizes the development of Duke Children’s multidisciplinary care team and their dedication to providing the best care and services for people and families living with Duchenne.
Breaking News! FDA Releases Draft Guidance for Duchenne
Today the FDA has released their draft guidance on Duchenne. This is the first time that a patient community has proposed and drafted a guidance for FDA to use as a jumping off point for their own drug development guidance an official map for how companies can move their Duchenne-focused innovation through the regulatory approval process.
Update on the FDA Guidance
Last week we met with FDA to discuss status and potential release of guidance to help accelerate development and review of potential therapies for Duchenne. PPMD President Pat Furlong provides an update from the meeting.
September 4-6, 2015 in Disneyland (California)