For the third year in a row, the global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This day, this simple idea, has blossomed into a truly incredible opportunity for families and friends affected by Duchenne to tell their neighbor, or co-worker,…

As we quickly approach World Duchenne Awareness Day, on September 7, I am pleased to provide details on our team’s ongoing trip to South Africa.

Over the past 10 years, the Muscular Dystrophy Foundation of South Africa (MDSA) has…

I have a confession…

I have had the privilege of serving as an advocate for families whose children have Duchenne within school systems for more than 20 years, have attended hundreds of IEPs and 504 Plan meetings, and have given even more school presentations, assemblies and in-services about Duchenne… but this year is completely different.…

Join us September 24th as thousands of coaches from hundreds of universities wear the Coach To Cure MD patch on their sleeve to raise awareness. Families will join teams on the field on game day and will share their stories with spectators and media. We also…

This past weekend marked the third and fourth stops on PPMD’s Every Single [One] Tour – one taking place at Denver Children’s (University of Colorado) – one of PPMD’s Certified Duchenne…

On Friday, PPMD submitted additional comments to the latest version of PDUFA VI.  At this point, there are few members of our Duchenne community who haven’t heard the acronyms ‘PDUFA’ or ‘FDASIA’. But, it’s possible that there are many among us who aren’t aware just how…

Last month, PPMD welcomed Dr. Eric Olson from UT Southwestern Medical Center to discuss the basics of gene editing and the potential permanent correction of Duchenne mutations with CRISPR/Cas9. If you missed it live, check out the recording to learn more about this technology and what…

We know that many within our community have long been advocating for and following the passage of The Achieving a Better Life Experience (ABLE) Act and subsequent IRS rules and state implementation. In June, the first state ABLE programs launched with the initiation of Ohio’s STABLE program. At …

Today, PPMD sent a letter to the FDA requesting updates on various regulatory actions of tremendous importance to the Duchenne community. In our letter, we are asking the FDA to talk to our community, tell us what is going on, and confirm that…

Last week you heard from my friend Kim Powers. She and her husband have jumped into this year’s 9th Annual Coach To Cure MD…

Marathon Pharmaceuticals announced this morning that the U.S. Food and Drug Administration (FDA) has accepted Marathon’s New Drug Applications (NDAs) for the investigational drug deflazacort for Priority Review. The NDAs are for an immediate-release tablet formulation and an oral suspension formulation of deflazacort for the…

Today, Summit Therapeutics plc announced positive results from a Phase 1 trial of a new formulation of…

The 9th Annual Coach To Cure MD on September 24 is shaping up to be the biggest year yet!

Duchenne mom Kim Powers shares why this is the year you should get in the game and help us #TackleDuchenne:…

On July 30^th, I joined the Ride4Gabe Team for the second leg of their eleven-day trek which started in Houlton, Maine and ends in Mobile, Alabama.  Representing Parent Project Muscular Dystrophy (PPMD) and as the father of two sons who have Duchenne muscular dystrophy, I rode 150-miles of the day’s 213-mile route.   By every measure, it was a great…