In an effort to answer some of the questions that have arisen with the potential FDA approval of deflazacort in the U.S., PPMD has worked with both Masters and Marathon to put together the resource below, which we hope will provide clarity.…

Patient Assistance programs are typically non-profit organizations sponsored by drug companies, patient advocacy organizations, and civic groups that establish disease specific programs to facilitate access to brand-name medications.

In the case of…

PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

The Center for Duchenne Muscular Dystrophy at the…

PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

The Center for Duchenne Muscular Dystrophy at the…

The journey has been long. From PTC Therapeutics' first presentation about targeting a ‘stop sign’ within the dystrophin mutation to PTC 124 to ataluren and now, Translarna. The journey has been a long one.

The team at PTC are pioneers, the company…

Solid Biosciences and its subsidiary, Solid GT, announced today that the FDA and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001,…

You are invited to join us February 12-14, 2017 in Washington D.C. for what is going to be another critical year in Duchenne advocacy.

2016 marked a tremendous…

This presentation, "The Emergent and Acute Management of Patients Living with Duchenne Muscular Dystrophy," was produced in an effort to educate clinicians in the care of individuals and families living with Duchenne.

The…

When I first joined the Duchenne community in 2009 at Children’s National Medical Center, I remember thinking that I was walking into a critical juncture in Duchenne history. There were three compounds in the clinic (drisapersen, ataluren, and eteplirsen aka Exondys 51)…

Join us for our first DuchenneConnect Twitter chat on Thursday, Oct 20, 2016 from 1-2:00 PM EST. We will be using the hashtag #DuchenneConnectTC.

Our first chat will cover the…

PPMD is disappointed to learn that the FDA denied PTC Therapeutics' first appeal of the refuse to file letter issued in February. As we understand it, this is a process and may involve additional appeals. PPMD will be…

It is hard to imagine anything worse than receiving the diagnosis of Duchenne. The dates and odysseys that brought us into this community differ, but the fervor we share to slow and stop the progression of this disease as it marches through our children is unparalleled in any…

Can you believe 2017 is just around the corner? It will be here before you know it and PPMD is busy putting together an exciting list of events that will continue to connect and inform our Duchenne community. It’s already a packed new year!

Check out where your PPMD family plans to be in 2017 and mark your calendars!…

Parent Project Muscular Dystrophy (PPMD) has awarded a $239,000 grant to Dr. Terence Partridge, Professor of Systematic Integrative Biology, Research Center for Genetic Medicine at Children's National Medical Center. This grant will help Dr. Partridge and his team continue their work in…

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…

Last week, PPMD began a webinar series on access – an exciting new chapter for our Duchenne community. But we are just at the beginning of this chapter and there is lots to explore.

We knew this day was coming – we…

PPMD is deeply troubled to learn that health insurer Anthem will not be covering Exondys 51, stating the following position:

“The use of eteplirsen is considered investigational and not medically necessary for all indications, including but not limited to the treatment of Duchenne muscular dystrophy.”

This is not an…

PTC Therapeutics, Inc. today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory people with nonsense mutation Duchenne. PPMD continues to urge the FDA to provide PTC with a full review and advisory committee meeting.…

Last week, Ann Lucas and I had the honor of presenting an Educational Breakout Session focused on Duchenne at the National Society of Genetic Counselors (NSGC) Annual Education Conference in Seattle. Lauren Morgenroth from TRiNDS also presented during the…