There has never been a moment like this in our Duchenne community. Today the FDA released briefing documents on the application for eteplirsen. It is clear that we continue to face a serious regulatory challenge as a community.…
We know that many of you have questions regarding Marathon Pharmaceuticals and their expanded access program for deflazacort in the US. PPMD has reached out to the company to get answers to some of the questions. The FAQs below explain what…Continue
Added by PPMD on January 15, 2016 at 12:30pm — No Comments
The briefing documents for Sarepta’s Ad Comm, one week from today, have been released. We will spend the…Continue
Added by PPMD on January 15, 2016 at 11:46am — No Comments
The removal of “wisdom teeth” (third molars) is one of the most common procedures performed by oral and maxillofacial surgeons. While it is common, it is considered…Continue
Added by Kathi Kinnett on January 15, 2016 at 9:30am — No Comments
Today our community was dealt a painful blow as the FDA issued a Complete Response letter to BioMarin regarding the company’s New Drug Application (NDA) for Kyndrisa (drisapersen). As stated in BioMarin’s press release, “The FDA issues Complete Response letters to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. FDA…Continue
The Common Rule – or the 'Federal Policy for the Protection of Human Subjects' – is a human subjects biomedical and behavioral ethics rule that sets the ethical standards by which all…Continue
Added by Annie Kennedy on January 13, 2016 at 1:58pm — No Comments
In 2012, PPMD hosted a meeting with NHLBI entitled Contemporary Issues in Duchenne Cardiology. One of the small groups resulting from that meeting – the Animal Model Small Group – was tasked with…Continue
Added by Kathi Kinnett on January 13, 2016 at 1:00pm — No Comments
Next week, on January 22nd, an FDA Advisory Committee will review eteplirsen for potential approval and use within our Duchenne community.
The Duchenne community is joining forces in a show of strength to demonstrate to the FDA our community’s support for our robust therapeutic pipeline. All Duchenne community members are encouraged to attend the Advisory…Continue
Added by Annie Kennedy on January 13, 2016 at 11:30am — No Comments
Dysphasia, or difficulty swallowing, effects many patients with Duchenne. Complaints of "something stuck in my throat" are not uncommon. Symptoms may progress to difficulty swallowing saliva, coughing during and after…Continue
Added by Kathi Kinnett on January 8, 2016 at 9:23am — No Comments
PPMD supported the development of the HT-100 program from early on, including a $500,000 investment via our GIFTED program for the clinical trial. We are excited for the expansion of the clinical trial and thrilled for the relationship with Grunenthal. This incredible partnership enables Akashi to continue to be managed and led by parents and we are all hopeful HT-100 will be part of the cocktail of combined therapies that will achieve our highest priority--to…Continue
Added by PPMD on January 8, 2016 at 9:02am — No Comments
FibroGen announced enrollment of their first patients in an open-label, multicenter Phase 2 clinical trial of the investigational compound FG-3019 in non-ambulatory patients with Duchenne. We are thankful that FibroGen is addressing ways to treat fibrosis caused by Duchenne’s progression and are hopeful that this study will prove promising.
Learn more about FibroGen and FG-3019…Continue
Added by PPMD on January 7, 2016 at 10:00am — No Comments
Last month we told you we hoped to raise $200,000 to support new, early-stage research. Throughout the month, we told you about potential therapies like epicatechin. We told you about generous matches from Mary-Lou and Larry Weisman and the Dumm Family/…Continue
Added by Pat Furlong on January 5, 2016 at 11:00am — No Comments
Strategies that address the basic problem in Duchenne, the absence of dystrophin, are critically important to patients and families and have attracted considerable attention from drug developers.
We already have two technologies – exon skipping and stop…Continue
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
The European Medicines Agency (EMA) has just published guidelines for Industry on the clinical…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
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