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January 2016 Blog Posts (19)


Staff
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…

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


Staff
ACTION ALERT: Sign-On Extended! Urge House Member - Apply FDASIA Tools to Duchenne

Action Alert

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…

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Added by Ryan Fischer on January 25, 2016 at 11:30am — No Comments


Staff
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. …

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


Staff
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…

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


Staff
ACTION ALERT: Urge House Member - Apply FDASIA Tools to Duchenne

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.…

Action Alert

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Added by Ryan Fischer on January 15, 2016 at 2:45pm — 1 Comment

Gene Editing Offers Hope for Treating Duchenne Muscular Dystrophy, Studies Find

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…

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Added by William Locke on January 15, 2016 at 1:00pm — 1 Comment


Staff
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…

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


Staff
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…

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


Staff
Wisdom teeth & Duchenne

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…

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Added by Kathi Kinnett on January 15, 2016 at 9:30am — No Comments


Staff
A Blow to Our Community – But Our Work Continues

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…

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Added by Pat Furlong on January 14, 2016 at 12:30pm — 3 Comments


Staff
PPMD Responds to Revisions to “The Common Rule”

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…

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Added by Annie Kennedy on January 13, 2016 at 1:58pm — No Comments


Staff
Standard Operating Procedures for Duchenne Animal Models, Published

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…

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Added by Kathi Kinnett on January 13, 2016 at 1:00pm — No Comments


Staff
Duchenne History in the Making — and We Need You to Join Us! | January 22, 2016 | Silver Spring, MD

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…

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Added by Annie Kennedy on January 13, 2016 at 11:30am — No Comments


Staff
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…

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


Staff
Dysphagia in Duchenne muscular dystrophy: practical recommendations to guide management

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…

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Added by Kathi Kinnett on January 8, 2016 at 9:00am — No Comments


Staff
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…

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


Staff
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…

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


Staff
We did it! Over $270,000 raised to support early-stage research.

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/…

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Added by Pat Furlong on January 5, 2016 at 11:00am — No Comments


Staff
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…

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

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