SomaLogic Study Published: Another Piece of the Puzzle
Breakthrough news! Today, SomaLogic and PPMD - together with several other Duchenne research groups - published a significant discovery that explains a technology allowing researchers to accurately measure the individual proteins in very small amounts of blood or other samples. By comparing patient and control samples, identification of critical differences in protein concentrations can be identified rapidly. These significantly different proteins can then be used as the basis for developing new diagnostic and therapeutic approaches, including their use as biomarkers for quickly assessing the efficacy of promising new drugs. This is incredibly promising news for our community.
End Duchenne eNews: Connect - It's More Than Just a Conference
Catch up on the latest research and community updates in this month's End Duchenne eNews!
PPMD’s Connect Series: Bringing Together You & the Experts
We are excited to present the latest events in PPMD's Connect Roundtable hosted by our FACES groups and our Train the Trainers series! This summer and fall, PPMD will convene roundtables covering research updates and the latest care concerns, as well as physical therapy workshops to provide training to therapists and families… all in your own backyard! Check out the schedule for these exciting new Connect series events.
Newly Updated Vaccination Recommendations
As you know, PPMD works very hard to keep you as up to date as possible. Sometimes those updates come very quickly, as has recently happened with recommendations for vaccinations. While we just recently published a new “altered vaccination schedule,” discussions with the Centers for Disease Control and Prevention (CDC) have again updated the recommendations for vaccinations for people living with Duchenne.
PPMD's 2015 Connect Conference Agenda: Now Available!
PPMD is excited to share the agenda for our 21st Annual Connect Conference in Washington, D.C., June 18-21. Every year our agenda grows and expands to include more of the topics you and your family are the most interested in. This year is no exception, with more research, care, and advocacy updates than ever before!
Thank you for making the launch of PPMD’s Robotics Initiative such a huge success! In less than three weeks, we raised $165,400 to support innovative technology that we believe will benefit all people with Duchenne. Thanks to your generosity, we can support more projects and new work in assistive technologies. Thanks to you, PPMD can continue to look ahead at the full progression of Duchenne.
The “Let’s Get Them Moving” Workshop
PPMD joined Duchenne stakeholders from around the world in a special one-day meeting to discuss robotic technology and the potential it holds for our community. PPMD together with UPPMD gathered the best and brightest robotics experts and engineers who are working to apply their innovations to Duchenne. We discussed the variety of technologies currently in development technologies that inspired PPMD to launch our Robotics Initiative earlier this spring. Annie & John shared reports from the historic meeting.
BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Amenable to Exon 51 Skipping
The Duchenne community should celebrate. Today, Biomarin submitted the rolling NDA on drisapersen. This means we now have a second NDA submitted for Duchenne. Santhera is likely to follow soon. By first quarter 2016, we may see 3 approved drugs with more to follow. This is exactly what we have been working for, hoping for.
PPMD Awards $148,000 Grant to University of Washington
Exon skipping and gene replacement are leading candidate therapeutics in the Duchenne space right now. It is our hope that Dr. Froehner’s project will help improve decision making in the design and targeting of both exon skipping oligonucleotides and gene therapy vectors so that patients receive the best therapy possible and the chances of approvals in a timely manner are increased.
Webinar Recording: FOR-DMD Study
The recording of our webinar on the FOR-DMD study (developing standardized corticosteroid treatment for Duchenne) is now available. Included in the webinar is an overview of the aims of the FOR-DMD study, along with a discussion of why the study is particularly relevant in regard to current/future Duchenne clinical trials.
Webinar Recording & Summary: Orthopedic Complications in Duchenne
If you missed our webinar on Orthopedic Complications in Duchenne (covering contractures, scoliosis, & fractures), you can now access a recording of the webinar online. Summary notes and responses to additional questions we received during the webinar are also available.
Recapping the FDA-NIH Dystrophin Methodology Workshop
PPMD CEO, Dr. John Porter, recaps the FDA and NIH dystrophin methodology workshop. This was an important moment for the Duchenne community and PPMD was honored to be a part of it. Thank you to all the families who came to the meeting.