[Upcoming Webinar] A Closer Look at the Potential of CRISPR/Cas9 in Duchenne
Building on our introductory webinar on CRISPR/Cas9 that we held over the summer, join us Thursday, December 15 at 2:00 PM ET for our second webinar on this exciting technology.
Action Alert: Urge Senate to Vote ‘YES’ on 21st Century Cures Act!
Please contact your two US Senators today with one simple message—vote YES on 21st Century Cures!
Your gift to CRISPR will be Matched!
There has never been so much momentum in this community. Help us seize this moment by donating toward CRISPR/CAS9 research-- every gift will be doubled!
A truly thankful moment
PPMD is so thankful for this community.
[Webinar Recording] NS Pharma's Exon 53 Skipping Program
PPMD recently hosted webinar with NS Pharma (US subsidiary of Nippon Shinyaku, Co.,Ltd) to discuss NS Pharma's Exon 53 Skipping Program.
[Webinar Recording] Deflazacort Access: Navigating Potential Changes to Your Prescription
PPMD recently hosted a webinar with Masters Pharmaceuticals to discuss the transition from Masters’ deflazacort to Marathon Pharmaceuticals deflazacort, should Marathon receive FDA approval in the U.S.
End Duchenne eNews: The Challenge of Gratitude
Catch up on the latest research and community updates in this month's End Duchenne eNews.
Duchenne Carrier Study at Nationwide Children’s Hospital
PPMD is excited to support this important study that will evaluate the effects of a lower level of dystrophin in female carriers. We hope the results of this study will help us better understand the implications of being a Duchenne carrier, and possibly apply some of the answers to our understanding of Duchenne in males.
Registration for the 2017 Bank of America Chicago Marathon is now open!
Join PPMD's Run For Our Sons team for the 40th anniversary of this incredible race, on Sunday, November 4, 2017. Our entries are only available until January 18th, so sign up soon!
Understanding Insurance Determinations
PPMD’s Annie Kennedy provides an update on our Duchenne community’s current access environment, and actions that those who are amenable to exon 51 skipping can take in order to encourage their providers to cover EXONDYS 51.
Deflazacort Access: Navigating Potential Changes to Your Prescription
PPMD has put together a resource we hope will answer some of the questions that have arisen regarding the potential FDA approval of deflazacort in the U.S.
The Assistance Fund Launches Duchenne Program to Support Access to Exondys 51
The Assistance Fund has launched a new program in Duchenne to support access to Exondys 51 after efforts through SareptAssist have been other-wise exhausted.
PPMD Designates Center for Duchenne Muscular Dystrophy at UCLA, a Certified Duchenne Care Center
Our Duchenne families in Los Angeles are extremely fortunate to have this team of dedicated clinicians and researchers at UCLA.
The Latest Update on Translarna
PPMD continues to believe that our Duchenne community deserves a full and fair review of the Translarna data by scientific experts at an Advisory Committee Meeting. If you have any experience on Translarna, or are waiting for access to Translarna, please share your experience with us so that we can translate your story into rigorous data that can be shared with FDA.
Register for PPMD's 2017 Advocacy Conference
Your presence in Washington, DC this February is a critical part of the Duchenne community's strength and impact going forward. Please consider joining us February 12-14 as we urge Congress to continue to make Duchenne a priority!
Duchenne Research Update: October 2016
So much has happened in recent weeks in the Duchenne space that it can be hard to keep up. PPMD’s Abby Bronson, SVP, Research Strategy provides a roundup of the latest Duchenne research news.
There has never been so much momentum in the Duchenne community. Help us keep moving forward by donating to fund CRISPR/Cas9 gene‑editing technology. Every gift to PPMD is being doubled until we raise $250,000—but we have to reach this goal by 12/31!