An Important Webinar Opportunity:

Since the passage of the ABLE Act in December of 2014 there has been a great deal of progress both at the state and federal levels. This progress includes the passage of over 35 state ABLE related pieces of legislation, the development of proposed rules and regulations, formal direction from the Social Security Administration, and a major amendment to the federal statute.

Given all this exciting progress, the…

PPMD and a coalition of Duchenne foundations continue to work together on preparing for the Sarepta Ad Comm. As of today, we are still waiting on a new confirmed date. With the postponement of the Ad Comm, the Congressional letter to FDA remains open. Has your House Members signed on…

Today, Catabasis Pharmaceuticals announced positive top-line results from Part A of the MoveDMD trial, a Phase 1 / 2 trial of CAT-1004 for treating Duchenne. The trial demonstrated favorable safety, tolerability, and pharmacokinetics in patients. These results support initiation of Part B of the trial expected in the first half of this year. This is promising news! PPMD is proud to have sponsored this trial early on and to be a partner with Catabasis. …

We are deeply saddened, disappointed, and distressed to report that the highly-anticipated Ad Comm for eteplirsen has been postponed due to the blizzard anticipated to hit DC on Friday. We are grateful to our extraordinarily powerful Duchenne community who has been willing to travel into the eye of this storm to fight Duchenne. Our community has proven once again to be a force stronger than any winter storm, and much stronger than Duchenne itself. As soon as we receive any updates…

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.…

Hi,

I'm an RN that works at Duke University Hospital in Durham, NC and ran across this article being promoted at my institution. I had recently signed Tonya's petition on Change.org so I wanted to post this information for those who may not have heard the news. It looks very promising! I'm not sure what could be done to possibly advance the research process in order to bring clinical trails closer to reality, but figured there may be those in this community that might have possible…

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 we have learned so far. As we learn more about Marathon,…

The briefing documents for Sarepta’s Ad Comm, one week from today, have been released. We will spend the weekend reviewing all of the materials the FDA has released. PPMD…

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 surgery. The questions of whether this is a safe surgical option and…

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…

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 government–funded research in the U.S. are held, and which are upheld by…

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 developing/updating the …

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…

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…

Dysphagia, 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 meals, increased meal duration, difficulty starting swallowing, loss…

NPR recently highlighted how the ABLE program may benefit Americans living with disabilities and helped provide further context for this quickly evolving resource. Click here to listen to this week's segment.

PPMD has long been involved in efforts to see the dream of ABLE accounts become a reality – and while not the…

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…

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/…

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 codon read-through therapy – that allow the body to…