News Update

Action Alert: Urge Congress to Vote ‘YES’ on 21st Century Cures Act This Week!

Please contact your Senators and Representative TODAY and let them know that THIS version of the 21 Century Cures Act is a game-changer for you. Urge them to vote ‘YES’ on the 21st Century Cures Act this week!
November 28, 2016 | Learn more 

News Update

A truly thankful moment

PPMD is so thankful for this community.
November 24, 2016 | Learn more 

News Update

[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.
November 22, 2016 | Learn more 

News Update

[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.
November 18, 2016 | Learn more 

News Update - eNews

End Duchenne eNews: The Challenge of Gratitude

Catch up on the latest research and community updates in this month's End Duchenne eNews.
November 15, 2016 | Learn more 

News Update

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.
November 14, 2016 | Learn more 

News Update

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!
November 2, 2016 | Learn more 

News Update

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.
November 2, 2016 | Learn more 

News Update

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.
October 31, 2016 | Learn more 

News Update

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.
October 28, 2016 | Learn more 

Certified Duchenne Care Center

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.
October 26, 2016 | Learn more 

News Update

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.
October 26, 2016 | Learn more 

News Update

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!
October 20, 2016 | Learn more 

News Update

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.
October 18, 2016 | Learn more 

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Let's seize this moment.


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!