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…

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…

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…

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…

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…

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…