Throughout the history of Duchenne, there have been moments that we recognize as inflection points, times when we saw a significant shift in the fight to end Duchenne, when the…

Solid Biosciences announced today that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne.

The Phase I/II adaptive study, called IGNITE DMD, will evaluate the safety and efficacy of a single intravenous (IV) dose of SGT-001 in ambulatory and non-ambulatory adolescents and children with Duchenne. Enrollment will begin at the first clinical trial site in the United…

Capricor Therapeutics today announced that the FDA has cleared its Investigational New Drug (IND) application to conduct a new clinical trial of CAP-1002, its lead investigational therapy, in boys and young men in advanced stages of Duchenne. PPMD looks forward to being join by Capricor for a community webinar tomorrow, November 30 at 1:00 PM EST…

On November 4, PPMD had the pleasure of working with the parents and providers of New Mexico to present our final Every Single One Tour stop of 2017 (click here for a preview…

Navigating access to approved therapies can be both time consuming and daunting. The time between approval by FDA to the day of a patient’s first commercial dose can vary depending on which insurance you have, your age and…

Earlier this month, I testified on behalf of PPMD and the national Duchenne community before the Advisory Committee on Heritable Disorders in Newborns and Children – the U.S. federal…

What an odd year it has been. I won’t say it has been a bad year, not entirely, but it has been dark. And I don’t think it’s just me.



Even social media, something I turn to for connection, a familiar friend, a warm embrace…has felt toxic. So many Facebook…

PPMD is excited to learn that Capricor's investigational cell therapy, CAP-1002, is showing significant improvements in people with Duchenne. We look forward to learning more details about this therapy in a community webinar on Wednesday, November 29 at 1pm eastern. Details coming soon!

Read the release from…

Call & Email Your Senators Today!

Both the House & Senate have now released their Tax Reform proposals, each with variations of similar policy impacts and changes that we feel an obligation to alert you to. It is anticipated that both Chambers will move this legislation swiftly and work to complete their votes during the week…

PPMD has been continuing to engage with both PTC and the FDA on behalf of the patient community regarding the regulatory review of ataluren.

Earlier this month, …

Any updates on HT-100?

Progress! Today, Anthem issued a revised policy regarding Sarepta’s EXONDYS 51. In the revision, Anthem declared EXONDYS 51 medically necessary for the treatment of Duchenne muscular dystrophy when all of the following criteria are met:

1. Individual has a confirmed diagnosis of Duchenne muscular dystrophy; and
2. Individual must be ambulatory (with or without needing an…

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?

Hear first-hand what…

Wave Life Sciences Ltd. announced the initiation of a global Phase 1 clinical trial for WVE-210201 in Duchenne patients amenable to exon 51 skipping. PPMD is excited by the progress Wave Life Sciences has made in exon skipping. While advances have been made in exon skipping, especially recently, there is more to explore within this technology. We…

More good news in gene therapy! Today, Sarepta Therapeutics announced that Nationwide Children’s Hospital has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for the micro-dystrophin gene therapy program. The Phase 1/2a clinical trial, now open to enrollment and scheduled for initiation by mid-November, is designed to assess the safety and tolerability of AAVrh74.MHCK7.micro-Dystrophin in individuals…

Call & Email Your Senators Today!

Yesterday, the Senate released the Tax Cuts and Jobs Act which includes many issues that we feel an obligation to alert you to.

While PPMD does not typically engage in legislation related to tax reform, this proposal (being referred to as…

Once again, PPMD is excited to see industry, patient groups, and research partnering, as today Nationwide Children's Hospital and Sarepta Therapeutics announced the achievement of an important milestone in…

Yesterday, the New England Journal of Medicine (NEJM) published “Single-Dose Gene-Replacement Therapy for Spinal Muscle Atrophy.” 

Spinal muscular atrophy (SMA)…

I decided to join the PAAC after Parent Project’s 2015 Annual Connect conference in Washington, D.C. Annie, along with the 2015 PAAC members and a few other adults with Duchenne, hosted a pre-conference meeting for teens and adults living with Duchenne. When listening to…