Masters now has an increasing supply of Deflazacort (a steroid available to treat Duchenne outside the US). However, due to recent regulatory changes, families should now allow 3…

Presentations from Day 3 of PPMD’s 21st Annual Connect Conference in Washington, D.C. are now available for download. If you were unable to attend this year’s conference or if you want to replay a presentation that you found particularly useful, we have been sharing all of the presentations online over the past couple of weeks. Check out the presentations from Day 3…

Since the enactment of the Stephen Beck, Jr., Achieving a Better Life Experience Act of 2014 (ABLE Act) in December 2014, PPMD has been working with leading disability organizations to ensure that states adopt and effectively…

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Chances are your life will not go exactly as planned. Even with your best, it's a near certainty you will face headwinds, hardships, or obstacles along the way that force you to change course. If you haven’t experienced this already, just wait. For…

Grant Will Be Used to Support Pilot Study for X-Ar Exoskeletal Arm Technology

Parent Project Muscular Dystrophy has awarded Talem Technologies a grant for over $70,000 to support a pilot study of their…

Presentations from Day 2 of PPMD’s 21st Annual Connect Conference in Washington, D.C. are now available for download. If you were unable to attend this year’s conference or if you want to replay a presentation that you found particularly useful, we will be sharing all of the presentations over the next couple of weeks. To start, check out the presentations from Day 2…

The first batch of presentations from PPMD’s 21st Annual Connect Conference in Washington, D.C. are available for download. If you were unable to attend this year’s conference or if you want to replay a presentation that you found particularly useful, we will be sharing all of the presentations over the next couple of weeks. To start, check…

“We are the population that was hoped for — and now we’re here.”

- L. Vance Taylor



This statement made by a close friend of mine perfectly captures the spirit that satiated the room at the PPMD Duchenne/Becker Transitions Meeting which took place at this year's Connect Conference. For two days, teens, young adults, parents,…

PPMD’s 2015 Connect Conference booklet contained over 40 pages of information on what’s currently going on in Duchenne research & clinical trials. We encourage you to browse the digital version of the booklet if you were unable to attend. And stay tuned for more research updates as we'll be posting recordings of presentations from the conference over the next few…

21 Century Cures. First a bold concept led by two visionary congressionary leaders on April 30th, 2014.

Then innovative ideas poured in to Washington from the world’s best and brightest developers and innovators.

Then lots of Hearings, and roundtables, and Hill meetings, and constituent visits.

Then ‘provisions’ were structured which pulled…

For over a year, PPMD has been working with Congress on landmark legislation called 21st Century Cures. The bill aims to speed the discovery, development, and delivery of therapies for diseases that have an unmet medical need like Duchenne. We have been thankful to Congress for their willingness to meaningfully engage the patient…

PPMD is excited that Catabasis has received Fast Track designation for CAT-1004 and we are proud to be an early supporter of this promising therapy.

Catabasis Pharmaceuticals Receives FDA Fast Track Designation for CAT-1004 for the Treatment of Duchenne Muscular Dystrophy

CAMBRIDGE, MA, July 6, 2015…

July marks the 25th Anniversary of Americans with Disabilities Act (ADA), 50th Anniversary of Medicaid, and the dawning of a new age therapy development for our Duchenne community. But - as we all know - with new opportunities, come new challenges. As people with Duchenne grow up and grow into adulthood, barriers to 'independence' are found in our federal/ state/…

We have a bedtime routine, Alex and I, a bonding conversation that has happened every night, without fail, since he has been about three years old. It's a tender moment between father and son, one where Alex shares his hopes and fears, and I’m fortunate to see life from an unique perspective. He’ll tell me about a girl he likes or a disagreement with a friend. He’ll tell me about a new video game that’s coming…

Historically, people with Duchenne have been told that using inhaled anesthetics can cause malignant hyperthermia (“MH”; extremely high body temperature), a condition causing rhabdomyolysis (breakdown of skeletal muscle tissue causing the release of myoglobin, a muscle protein that damages the kidneys) and…

There are many instances and outcomes that parents do not wish to consider.  The first is that one of their children will be diagnosed with a terminal illness, and one where there is no cure.  The loss of a child is a sorrowful event but standing with them as a caregiver in the face of a relentless disease progression is heart breaking.   There are no words to describe the…