This is it. We’re so close. We’re just $24,000 away from our goal of $200,000 to invest in early-stage research that can help the entire community.
And we’re also just a few short hours until our matching gift deadline. …Continue
Added by PPMD on December 31, 2015 at 8:00am — No Comments
Two weeks ago I came to you with exciting news and a challenge: The Dumm family had stepped forward to offer a second match for this year’s holiday campaign, after a generous match from the Weismans.
Well here we are, less than 48 hours away from a New Year and just…Continue
Added by PPMD on December 30, 2015 at 8:00am — No Comments
This is my favorite time of year. Not because of the presents or the new Star Wars movie—which by the way is AMAZING!—but because it feels like there is hope in the air.
A couple of weeks ago, when my parents finished listening to a webinar, they were so excited.…Continue
Added by PPMD on December 28, 2015 at 9:58am — No Comments
I didn't deal well with my son's diagnosis of Duchenne, at first -- I took a selfish, cowardly, escapist approach, numbing my mind with sex, drugs, alcohol, cigarettes and other self-destructive addictions. I let my son's disease feed my disease; it didn't do a…Continue
The European Medicines Agency (EMA) has just published guidelines for Industry on the clinical investigation of medicinal products for the treatment of Duchenne and…Continue
Added by PPMD on December 23, 2015 at 9:57am — No Comments
The holidays are an exciting time for our family—as I’m sure they are for yours. We’re grateful to be spending Christmas with our entire family. We take advantage of every holiday, every birthday, every special occasion to celebrate the strength of our family. And we never take for granted the time we have been given to be…Continue
Added by PPMD on December 22, 2015 at 3:00pm — No Comments
Earlier this month, PPMD announced a partnership with BIO to develop a set of general guidelines and recommendations that stakeholders can use to design and execute disease-specific patient preference studies. With PPMD's history designing and conducting patient preferences studies in Duchenne, we believe strongly that our…Continue
Added by PPMD on December 22, 2015 at 10:30am — No Comments
Added by John D. Porter, PhD on December 21, 2015 at 11:03am — No Comments
BioMarin has announced that the FDA will not make a decision regarding drisapersen until early January 2016. PPMD will continue to help both BioMarin and the FDA any way we can over the next few weeks. Thank you to the families who are spreading awareness about their experiences on drug. We will keep you updated on all developments.
BioMarin Announces That FDA Has Advised it Will Not Take Action on the Kyndrisa™…
Added by PPMD on December 18, 2015 at 8:30am — No Comments
A recent paper by Michael Rudnicki’s group at Ottawa Hospital Research Institute and the University of Ottawa has provided new insights into what we view as the critical targets for therapies in Duchenne (Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division. Dumont et al., Nature Medicine 21 (12): 1455-63, 2015). …Continue
Added by John D. Porter, PhD on December 17, 2015 at 4:00pm — No Comments
PPMD's Annie Kennedy has been actively representing our Duchenne community as discussions around the next version of the Prescription Drug User Fee Act (PDUFA VI) have begun between the FDA, industry, and the patient advocacy community. Today, Annie testified before the FDA as to our…Continue
Added by PPMD on December 17, 2015 at 3:27pm — No Comments
We have exciting news—and even more exciting news!
We are just over halfway to our $200,000 goal—thanks to so many of you donating and the generous matching gift from the Weismans!…
Added by PPMD on December 17, 2015 at 11:00am — No Comments
In the wee hours of this morning, Congressional leaders released the text of a massive spending bill (called the “Omnibus Spending Package”) that will fund the government for the remainder of Fiscal Year 2016, which runs through the end of September 2016. PPMD is very pleased that this bill contains a number of provisions that will benefit the Duchenne…Continue
Added by PPMD on December 16, 2015 at 2:00pm — No Comments
We wanted to take a moment today and let you know where things stand from PPMD’s perspective since the drisapersen Advisory Committee Meeting on November 24 (click here…Continue
Added by PPMD on December 15, 2015 at 3:24pm — No Comments
When Larry and I lost our son Peter to Duchenne more than 35 years ago, there was virtually no Duchenne community to speak of. We felt completely alone.
So much has changed in the ensuing years. To begin with, PPMD was founded. And it’s no accident that, since then, we’ve…Continue
Added by PPMD on December 15, 2015 at 10:25am — No Comments
There has been new information regarding the March 2015 recall of Deflazacort batches JL21 and JL28 from Masters. It seems that these batches were recalled due to a suspected lack of stability of the drug when stressed by time, temp, etc.…Continue
Added by PPMD on December 14, 2015 at 1:00pm — No Comments
Michelle Ioannou took part in Giving Tuesday in honor of her brother Christopher, 21, who has Duchenne! To date she has raised $2,600 through her own social media and email outreach. PPMD is honored to be the…Continue
Added by PPMD on December 10, 2015 at 4:00pm — No Comments
In October, PPMD held a FACES Roundtable in Houston that included an incredible line up of global experts in care and research. In an effort to ensure this information reached families beyond Houston, we recorded the sessions. Special thanks to Rachel and James Poysky for pulling together such a wonderful event.
Stay tuned for more roundtable…Continue
Added by Ryan Fischer on December 10, 2015 at 3:30pm — No Comments
For over twenty years, the Food and Drug Law Institute (FDLI) has presented the Distinguished Service and Leadership Award to individuals for their sustained service and leadership or exceptional contribution to the…Continue
Added by PPMD on December 10, 2015 at 9:30am — No Comments
Today, the Biotechnology Industry Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) announce the launch of a new initiative designed to share best practices for the development of disease-specific patient preference studies. Through this effort, BIO and PPMD will produce a document…Continue
Added by PPMD on December 9, 2015 at 2:08pm — No Comments