PPMD's Blog – January 2016 Archive (9)

Learn More About ABLE Implementation in 2016

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…


Added by PPMD on January 27, 2016 at 10:59am — No Comments

Catabasis Pharmaceuticals Announces Positive Top-Line Results from Part A of the MoveDMD Trial, a Phase 1 / 2 Trial of CAT-1004 for Duchenne

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. …


Added by PPMD on January 25, 2016 at 9:30am — 1 Comment

FDA Ad Comm for Eteplirsen Postponed Due to Inclement Weather

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…


Added by PPMD on January 20, 2016 at 7:09pm — No Comments

Marathon Expanded Access Program for Deflazacort: FAQs

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,…


Added by PPMD on January 15, 2016 at 12:30pm — No Comments

Briefing Documents for Sarepta’s Ad Comm Released

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…


Added by PPMD on January 15, 2016 at 11:46am — No Comments

Akashi Therapeutics Establishes International Partnership with Grünenthal Group on HT-100 for DMD

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…


Added by PPMD on January 8, 2016 at 9:02am — No Comments

Several States Poised To Offer Savings Accounts For Disabled Americans

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…


Added by PPMD on January 7, 2016 at 12:17pm — No Comments

FibroGen Enrolls First Patients in Phase 2 Clinical Study of Anti-Fibrotic Drug Candidate FG-3019 for the Treatment of Duchenne Muscular Dystrophy

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…


Added by PPMD on January 7, 2016 at 10:00am — No Comments

Finding Hope in CRISPR/Cas9

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…


Added by PPMD on January 4, 2016 at 1:30pm — 5 Comments

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