All Blog Posts (1,722)

Let’s say that Joe has Duchenne. If Joe breaks his arm and the EMT comes before Joe’s parent arrives, Joe may not remember to tell the EMT that: 1) he has Duchenne, 2) he is on daily steroids 3) he is allergic to penicillin and, in that painful scary moment, he may or may not remember his parent’s…

As you know by now, September is Duchenne Action Month. And…

The Senate is considering another effort to repeal the Affordable Care Act (ACA) before the end of the month. Last week, Senators Lindsey Graham (R-SC), Bill Cassidy (R-LA), Dean Heller (R-NV), and Ron Johnson (R-WI) released a new plan to repeal and replace the ACA. This proposal is commonly referred to as…

Everyone, six months of age and older, needs to be vaccinated against influenza (flu) by the end of October, if possible. Protecting people living with Duchenne is especially important. A …

Santhera Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion for its Type II extension application for Raxone® (idebenone) in Duchenne. Santhera plans to appeal the opinion and will seek a re-examination by the CHMP. We appreciate…

Today Parent Project Muscular Dystrophy (PPMD) named Nemours/Alfred I duPont Hospital for Children (Nemours) in Wilmington, Delaware our 17th Certified Duchenne Care Center.

PPMD’s Certified Duchenne Care Center Program,…

There are some days when I feel like our community is in its infancy. When I look at where we are in terms of access, therapeutic options, combinations, care implementation…



But more often than not, I am overwhelmed with gratitude for the infrastructure and resources…

We kicked off our fall Every Single One Tour stops in West Virginia this past weekend! We appreciate all of the families, speakers, and volunteers that spent the day with us in…

Paperwork – oh the paperwork!

Each time you visit a physician’s office or hospital, we leave with piles of forms and papers detailing the visit. The providers seen, the prescriptions written, results from tests conducted, instructions for follow up, and more. And each of those pieces of…

The deadline to submit a written comment for the FDA Advisory Committee Meeting on ataluren is this upcoming Thursday, September 14th.

To make a…

The dynamics of weight loss are intriguing, in take a look at the many because they came from desire to shed some weight in comparison to the small number who do. You would think with all the diet plans and dietary supplements, that weight problems would go away. But that hasn't came about, so something must be wrong. The sheer number of programs and products out there helps it to be difficult to find out what is effective and what is not. And it is far from as if one solution is effective…

PPMD is excited to announce next year’s lineup of areas we will visit as part of our End Duchenne Tour!

Combining each of the pillars that make up PPMD’s mission, the End Duchenne Tour brings updates on research, advocacy, and care to cities across the…

Earlier this year, Bristol-Myers Squibb (BMS) announced it had entered into an agreement to license BMS-986089 (RG6206), an anti-myostatin adnectin in development for Duchenne muscular dystrophy, to Roche. Today, the Roche team has…

Yesterday, Kymriah, a gene therapy made by Novartis that treats an aggressive type of leukemia was approved for children and young adults. The new commissioner of the FDA, Dr. Scott Gottlieb,…

Every year at PPMD's Annual Connect Conference, we try to put together the best topics, with the most informed speakers, to address elements of care at…

With trials in gene therapy on the horizon, and terms like "cassette", "construct", "promoter", and "transduce" being thrown around, we want to ensure that you, our Duchenne Community, has all the knowledge you need to navigate the world of gene therapy.

Earlier this…

The Duchenne community is now preparing for the September 28 Advisory Committee Meeting for PTC’s therapy ataluren. Whether submitting written…

May 12-13, 2016, PPMD convened a workshop of 48 opinion leaders, research experts and clinicians from the US and Canada, on the subject of Duchenne and bone biology. Bone health has long been a…

Hope is critical to survival. Hope helps us get through those times of darkness with the thought that there is something new on the horizon, a potential new destination. Hope enables us to focus on meeting challenges even in the midst of adversity. Hope allows us to see that there might be…