PPMD Designates Yale New Haven Children's Hospital a Certified Duchenne Care Center
PPMD is excited to announce our 13th Certified Duchenne Care Center (CDCC) the Yale New Haven Children's Hospital's Muscular Dystrophy Program!
[Upcoming Webinar] Summit's Utrophin Modulation Program
Please join PPMD and Summit on Wednesday, January 25 at 11:00 AM ET for an educational webinar on utrophin modulation, an approach for Duchenne that has the potential to treat all patients.
PPMD Awards $600,000 Grant to NJIT & Talem for Next Phase of Upper Extremity Exoskeleton
PPMD has awarded a $600,000 grant to the New Jersey Institute of Technology (NJIT) and Talem Technologies as part of our ongoing exploration of robotic technology to assist people living with Duchenne.
End Duchenne eNews: A New Year's Resolution
Catch up on the latest research and community updates in this month's End Duchenne eNews.
New Resource to Help Navigate EXONDYS 51 Access
Dr. Craig McDonald of UC Davis has shared data and an accompanying presentation to serve as a resource for families who have received an EXONDYS 51 denial from their insurance company and are working to appeal the decision. You should insist that both your clinician and insurance company review this data and presentation.
[Upcoming Webinar] Vamorolone (VBP15) Clinical Trials in Duchenne
Please join PPMD and ReveraGen BioPharma as we host a webinar Wednesday, January 18 at 1:00 PM ET to discuss vamorolone (VBP15) clinical trials in Duchenne.
New Research & Clinical Trial Tools on DuchenneConnect
We are excited to announce three new tools now available on PPMD's DuchenneConnect! These tools are meant to help you in your journey as you gather information around potential research studies and potential new exon skipping therapies.
PPMD Awards $2.2 Million Grant to Explore Gene Therapy in Duchenne
PPMD has announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital. This grant is the first project of a new Gene Transfer Initiative PPMD is launching this month that will include the work Dr. Mendell and Dr. Rodino-Klapac are doing, as well as a deeper exploration of CRISPR/Cas9 technology as a potential therapeutic approach to treating Duchenne.
Two simple words...THANK YOU!
We could not be more overwhelmed by the incredible response to our holiday campaign. Thanks to this community, we head into 2017 with incredible momentum—and the funds to move CRISPR/Cas9 research forward.
What You Need to Know about Duchenne and Viral Gastroenteritis (That Horrible Stomach Bug!)
Viral gastroenteritis — often called the stomach flu — isn't fun for anyone, but it is especially worrisome for a person living with Duchenne muscular dystrophy. Our latest blog explains what you should do if a person with Duchenne contracts the virus, as well as what you can do to stay as healthy as possible.
How PPMD’s Certified Duchenne Care Centers Are Improving Quality & Access to Care
People with Duchenne are being cared for by clinics both near and far. Every one of them deserves the best care and treatment possible. Learn how PPMD’s Certified Duchenne Care Center program is making comprehensive Duchenne care more accessible and available for our families.
Understanding the Path to Access -- Resources for Families
We have all had frustrations getting healthcare paid for.
Coverage can be especially difficult when new medicines or procedures are recommended. PPMD has assembled resources to help families and medical providers at each stage of the healthcare access process.
Webinar Recording: A Closer Look at the Potential of CRISPR/Cas9 in Duchenne
PPMD recently hosted a webinar with Dr. Eric Olson from the Department of Molecular Biology at UT Southwestern Medical Center to review the basics of gene editing and next steps in the research and development potential of CRISPR/Cas9 in Duchenne.
The Killian Family Will Match Your Gift!
The Killian family is matching every online donation to PPMD until we’ve raised $250,000! Your gift will help PPMD fund important research into CRISPR/Cas9, but we have to reach this goal by 12/31—please donate today!
PPMD Is Seizing This Moment to Explore the Potential of CRISPR/Cas9 in Duchenne
CRISPR/Cas9 gene-editing technology shows incredible promise to target and modify DNA. PPMD is working with researchers to better understand the potential of CRISPR/Cas9 technology in Duchenne.
BREAKING NEWS: 21st Century Cures Crossed Over the Finish Line!
To everyone in our PPMD community who called, reached out, emailed, and met with your elected officials -- WE DID IT!!
We are thrilled that Congress has passed the 21st Century Cures bill which includes almost every provision that our Duchenne community championed and led throughout this process.