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.
End Duchenne eNews: PPMD Launches Robotics Initiative
Catch up on the latest research and community updates in this month's End Duchenne eNews!
We See Strength in Innovation
Innovation. We are blessed in the Duchenne community to have forward thinking geniuses, creating devices that we believe will help spare muscle deterioration in people with Duchenne. Engineers continue to make huge strides in robotic interventions. That is why this month PPMD is launching a Robotic Initiative. Our goal is to raise $200,000 between now and May 1 so that we can provide grants to the most innovative technology in the field.
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.
PPMD Participating in FDA-NIH Dystrophin Methodology Meeting on Friday, March 20
PPMD is grateful that the FDA and NIH are willing to discuss dystrophin and have opened their doors to include our community leaders.
If you would like to attend Friday’s public scientific workshop, or watch a live stream of the meeting, view details here.
Dollars Destroying Duchenne
PPMD loves creative, out-of-the-box fundraisers that raise awareness. This new, annual event from Davenport, FL is a great example of something you can do in your community. Read organizer Diana Rodriguez’s story and don’t hesitate to reach out to us with your own ideas!
PPMD's 2015 Connect Conference Registration Open
This year, 2015, marks our 21st Connect Conference. With the Duchenne landscape changing in so many ways, this year's meeting will help you understand the nuances of Duchenne including the latest in research and care, open communication channels between thought leaders in the field, and lay the groundwork for future collaborations.
Spring Forward: Bringing the MD-CARE Act to Life
After months of outreach to congressional champions and visits to Capitol Hill, the MD-CARE Act Amendments were passed and signed into law in the end of 2014. Now the real work of implementation actually begins. On Tuesday, March 17, the MDCC will be taking the first formal step to bring the MD-CARE Act Amendments to life and our community will officially Spring forward!
What is Epicatechin?
Epicatechin. It’s a word that is starting to pop up more and more in the Duchenne community. But was is it? PPMD helps explain this exciting potential therapy now in the early stages of development.
Recall Notice for Deflazacort 30mg
If you receive this recall notice and/or think that you may have an affected batch (J021 [expiry 30/06/2017] or J028 [expiry 30/09/2017]), you should contact your provider and ask them to replace it with a prescription for prednisone to take until you get your new batches of deflazacort. Do not take deflazacort that is part of the affected batch. As more information becomes available, we will share it with the community immediately. If you have any further questions call your neuromuscular provider.
PPMD believes in supporting innovative ideas that will both improve daily living and maintain strength for those living with Duchenne. We hope you share our vision and will show your support of this exciting new technology by making a donation to PPMD’s Robotic Initiative today.
Make your tax-deductible donation and help us reach our goal by May 1.