June 2015 Blog Posts (15)

FDA Accepts BioMarin's NDA for Drisapersen; Sarepta Completes NDA for Eteplirsen

Big news in exon skipping therapy today! BioMarin announced this morning that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen.

We are such a fortunate…


Added by PPMD on June 29, 2015 at 10:34am — No Comments

Webinar Recording: PF-06252616 (Anti-Myostatin)

On June 3, 2015, Dr. Michael Binks, Vice President for Rare Disease Clinical Research at Pfizer, presented information about the upcoming clinical trial of  PF-06252616. The webinar provides an overview of Pfizer’s approach to advancing the science of an anti-myostatin monoclonal antibody as an investigational compound for potential therapeutic use in muscle wasting diseases, such as Duchenne muscular dystrophy. Dr. Binks gives a presentation and answers submitted…


Added by PPMD on June 25, 2015 at 3:30pm — No Comments

Urge your Senators to Cosponsor PFIA (S. 1597) from Home!

PPMD has been honored to lead the Duchenne community in developing patient-focused tools like our draft guidance and our benefit/risk surveys to help accelerate the FDA review of Duchenne treatments. Just last week, FDA issued its landmark draft guidance on Duchene therapy development, a document heavily informed by the project submitted nearly one year ago and led…


Added by Ryan Fischer on June 19, 2015 at 1:00pm — No Comments


Humanitarian Appeal: A very rare Neuro muscular disease (Muscular Dystrophy LGMD D2 Type) which these three brilliant shinning stars of Pakistan are facing. At least Share their case as much as you can so that Govt of Pakistan give some serious response on the request of a brave parents of these children.

SUPPORT the Three children treatment campaign of Mr. Shahid Zubair's suffering from a rare Neuro muscular disease and are running against time to get medical treatment from…


Added by www.Miracleforthree.com on June 16, 2015 at 1:50am — No Comments

Update on 21st Century Cures Initiative

Over the last year, we have been telling you about the 21st Century Cures Initiative, a comprehensive look launched by Congress to explore what could be done to accelerate the pace of cures in American therapeutic development.…


Added by Annie Kennedy on June 15, 2015 at 2:07pm — No Comments

Accessing Drugs Once They’re Approved

As the approval of therapies in Duchenne becomes more of a reality for our community, access becomes critical. Potential challenges with insurance coverage and approvals, as well as specialty pharmacy access begin to become a very real concern. And typically new and innovative therapies marketed to small patient communities are very expensive. Lingering…


Added by Annie Kennedy on June 15, 2015 at 1:30pm — No Comments

PPMD Listens

PPMD Listens. We have listened to patients, to families, to industry, and to providers. We have listened, and we have acted quickly and effectively, to help to improve your care.

  • In 2012, after listening to what care and services patients and families were not receiving, we convened the first Transforming Duchenne Care meeting.
  • In 2013, after…

Added by Kathi Kinnett on June 15, 2015 at 12:30pm — No Comments

From Where I'm Sitting: Using a Scooter for Mobility

          I told myself once that I would never write an article with this utterly corny and clichéd title, but as it turns out it works. It’s functional. It does exactly what I need it to do.

I ride a scooter to get around. I’ve been riding a scooter for 13 years now. That’s half my life at this point and I have no intention of switching. I use a scooter because it works for me. It’s functional. It does exactly what I need it to do. Kind of like the title of this…


Added by Buddy Cassidy on June 11, 2015 at 2:37pm — No Comments

PPMD's 2015 Connect Conference: Thank You to Our Sponsors!

Today, we are blessed with an overwhelming number of industry and corporate partners who have committed themselves to the fight to end Duchenne. This kind of commitment was unheard of when PPMD started 21 years ago. Because of the dedication of these companies and others, we are stronger as a community. We are closer to ending Duchenne.…


Added by PPMD on June 11, 2015 at 10:30am — No Comments

FDA Releases Draft Guidance for Duchenne

Amazing things happen when this community works together, when our messages are consistent, and our goals are in sync. We work together, we change the landscape, we make history.


Today the FDA has released their draft guidance on Duchenne. This is the…


Added by Pat Furlong on June 9, 2015 at 9:00am — 2 Comments

PPMD's 2015 Connect Conference: Kids Track - Mad Science & Sib Shop Program

One of our most exciting and fast-growing programs at PPMD's Connect Conference is our Kids Track! This year, we will be expanding our…


Added by PPMD on June 4, 2015 at 12:30pm — No Comments

Update on the FDA Guidance

Last week we met with FDA to discuss status and potential release of guidance. As you know, we submitted draft guidance to FDA in June, 2014. FDA posted the guidance for public comment for a…


Added by Pat Furlong on June 3, 2015 at 12:00pm — No Comments

PPMD’s Benefit/Risk work highlighted in groundbreaking reports

On May 13th the Medical Device Innovation Consortium (MDIC) released a report on incorporating patient preferences into the FDA’s benefit/risk assessments. It was much anticipated by PPMD as we continue our “aggressive engagement” with the FDA and industry trial sponsors. The report is full of really…


Added by Holly Peay on June 2, 2015 at 1:00pm — No Comments

The Duchenne Dash - Andew Elder from Masters cycles to Paris!

Andrew Elder, a Non-Executive Director on Masters Board, will be participating in the Duchenne Dash on 5th June 2015, in aid of The Duchenne Children’s Trust, cycling from London to Paris, a distance of over 200 miles, in 24 hours.

Andrew has been training hard for the event and is keen to raise awareness of this important cause.

Duchenne Muscular Dystrophy is a genetic condition causing muscle weakness.  It starts in childhood and usually affects boys…


Added by Rachel Skinner on June 2, 2015 at 11:03am — No Comments

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