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…
ContinueAdded by Abby Bronson on November 30, 2017 at 6:30pm — No Comments
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…
ContinueAdded by PPMD on November 30, 2017 at 10:24am — No Comments
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…
ContinueAdded by PPMD on November 29, 2017 at 8:30pm — No Comments
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 of our 2018 Tour schedule). We had the pleasure of…
ContinueAdded by Kathi Kinnett on November 20, 2017 at 9:00pm — No Comments
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 disease progression, and even where you live. With two…
ContinueAdded by Ryan Fischer on November 20, 2017 at 8:00pm — No Comments
Added by Annie Kennedy on November 20, 2017 at 6:30pm — No Comments
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 advisory committee on newborn…
ContinueAdded by Annie Kennedy on November 20, 2017 at 6:30pm — No Comments
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 posts are gas soaked, with every “Like” a potential flame.…
Added by Pat Furlong on November 16, 2017 at 1:00pm — No Comments
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…
ContinueCall & 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…
ContinueAdded by Annie Kennedy on November 15, 2017 at 1:46pm — No Comments
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, PPMD reached out to FDA requesting guidance on…
Added by PPMD on November 13, 2017 at 1:00pm — No Comments
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:
Added by Annie Kennedy on November 9, 2017 at 9:58am — No Comments
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 it's like to serve on the PPMD Adult Advisory Committee (PAAC) from current members…
Added by Annie Kennedy on November 8, 2017 at 2:30pm — No Comments
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…
ContinueAdded by PPMD on November 6, 2017 at 9:09am — No Comments
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…
ContinueAdded by PPMD on November 6, 2017 at 8:30am — No Comments
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…
ContinueAdded by Annie Kennedy on November 3, 2017 at 10:00am — No Comments
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…
ContinueAdded by PPMD on November 3, 2017 at 8:39am — No Comments
Yesterday, the New England Journal of Medicine (NEJM) published “Single-Dose Gene-Replacement Therapy for Spinal Muscle Atrophy.”
Spinal muscular atrophy (SMA) is a disorder caused by a mutation in the survival motor neuron gene 1…
ContinueAdded by Pat Furlong on November 2, 2017 at 9:54pm — No Comments
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 the other adults like me speak I felt a strong connection and saw myself fitting right in up there…
ContinueAdded by Colin Werth on November 2, 2017 at 2:21pm — No Comments
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