Different Together: PPMD Announces the Duchenne Regulatory Science Consortium
PPMD is thrilled to announce the launch of the Duchenne Regulatory Sciences Consortium (D-RSC) with the Critical Path Institute! Each instance of Duchenne seems to be as unique as the person it affects. The D-RSC will begin by aggregating a wide array of clinical data for analysis by consortium members, with the goal of developing a disease progression model that can be used in the development of new treatments, and reduce the amount of time it takes to get them to people living with Duchenne.
Changes to Deflazacort Ordering Process (via Masters Pharmaceuticals)
Masters now has an increasing supply of Deflazacort (a steroid available to treat Duchenne outside the US). However, due to recent regulatory changes, changes have been made to the ordering process to assure that the company is able to continue to supply Deflazacort to US families.
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.
September 4-6, 2015 in Disneyland (California)