Boston made for a great location for our October Every Single One Tour stop. Nothing beats the northeast in the fall. Boston is home to a number of great Duchenne organizations, a network of active families, and where most of the drug companies…

Ever wished you could change the world – or at least your piece of it? Been looking for a way to engage a bit more? Have personal experience that you’d like to see be better reflected in national policy and program efforts?

PPMD is seeking adult members of our Duchenne/Becker community to serve on the PPMD Adult Advisory Committee…

PTC has shared a letter with the Duchenne community in regards to the recent FDA decision. PTC will be holding a community call on Friday, October 27 at 12:00 PM EST to discuss next steps with the community. Please e-mail your…

Today the FDA issued a Complete Response Letter regarding PTC Therapeutics’ application for ataluren. Today’s news is hard. This decision is disappointing and not the outcome…

Lengthy trials with variable results, based on endpoints such as the 6-minute walk test that can limit trial enrollment to ambulant individuals with Duchenne – this is today’s reality for clinical trials in this disease.

But progress is being made and PPMD is proud of our role in changing the clinical trial process. Last week,…

In many diseases, the development of fibrosis in muscle, or any tissue, is the result of a reactive, or faulty tissue repair process involving many factors.

In Duchenne, fibrosis is present in skeletal muscles at a very early age and is one of the major factors contributing to the loss of muscle strength and…

PPMD thanks the 40 US physicians joining the call for approval of ataluren. This letter compliments the …

Do you ever wonder what happens to all the data we collect in the DuchenneConnect Registry? Ever wonder why we regularly ask you to update your registry account?

How your data is used:

Your data is used in many ways.…

On September 28 and 29, I had the privilege of attending an in-person Wellstone Center meeting that Dr. Eric Olson and his Wellstone team at UT Southwestern Medical Center hosted in Dallas, TX. The breadth and depth of what the Wellstone Centers have worked on over the years was apparent. I was struck by the number of therapies in the clinic…

Annie Kennedy and Kathi Kinnett from the PPMD team recently attended the 22nd International Annual Congress of the World Muscle Society and have been reporting back the latest updates related to…

PPMD applauds the group of 83 international experts with experience caring for Duchenne patients around the globe who have written a letter to the FDA encouraging approval of ataluren. With hundreds of patients accessing ataluren through…

Annie Kennedy and Kathi Kinnett from the PPMD team are attending the 22nd International Annual Congress of the World Muscle Society and will be reporting back over the next couple of days. More…

Steroid dosing in Duchenne can be a confusing topic – what is enough, what is too much, why weekend doses are higher. With these frequently asked questions I’ve heard from the community in mind, I reached out for input from Dr. Doug Biggar and Dr. Anne…

“WMS - What’s the buzz?”

It has been many years since I’ve had the opportunity to attend the World Muscle Society meeting and wow how things have changed! While PPMD has strong representation here every year, this year I have the pleasure of being here alongside Kathi…

Catabasis Pharmaceuticals, Inc. today reported new positive efficacy results following 24 and 36 weeks of treatment with 100 mg/kg/day oral edasalonexent in the MoveDMD Phase 2 trial open-label…

PTC Therapeutics President and CEO, Stuart Peltz, Ph.D has shared a letter with the Duchenne community following last week's Advisory Committee Meeting at…

This fall, PPMD is presenting a gene therapy webinar series with companies and institutions who are developing therapies for Duchenne that are commonly referred to as gene therapy, including…