Added by Annie Kennedy on January 13, 2017 at 11:00am — No Comments
The Duchenne Community’s Voice Was Essential Every Step of the Way & Contains PPMD’s Provision to Strengthen Patient Focused Drug Development Efforts
To everyone in our PPMD community who called, reached out, emailed, and met with your elected officials – WE DID…
Added by Annie Kennedy on December 7, 2016 at 2:30pm — No Comments
With the accelerated approval of EXONDYS 51™ (aka eteplirsen) by the FDA in September, the Duchenne community has been quickly introduced to a whole new vocabulary and a whole new list of concerns. We knew…
Added by Annie Kennedy on November 2, 2016 at 12:30pm — No Comments
Patient Assistance programs are typically non-profit organizations sponsored by drug companies, patient advocacy organizations, and civic groups that establish disease specific programs to facilitate access to brand-name medications.
In the case of…
Added by Annie Kennedy on October 28, 2016 at 11:00am — No Comments
With the initiation of 4 states ABLE programs already begun and Oregon having just anounced that their program will launch in December, any qualified individual (meaning most families with Duchenne) can open their accounts in 2016, if interested. We have previously shared information about the ABLE program and the…
Added by Annie Kennedy on October 13, 2016 at 8:37pm — No Comments
Ever wished you could change the world – or at least your piece of it? Been looking for a way to engage a bit more? Have personal experience that you’d like to see be better reflected in national policy and program efforts?
PPMD is seeking adult members of our Duchenne/Becker community to serve on the PPMD Adult Advisory Committee…Continue
Added by Annie Kennedy on October 12, 2016 at 11:30am — No Comments
Last week, PPMD began a webinar series on access – an exciting new chapter for our Duchenne community. But we are just at the beginning of this chapter and there is lots to explore.
We knew this day was coming – we knew that one day we would have our…Continue
Added by Annie Kennedy on October 11, 2016 at 3:00pm — No Comments
September is National Newborn Screening Awareness month and PPMD has been helping to put Duchenne on the newborn screening map — the WORLD map.
Our community understands the inherent benefits of early diagnosis. We know that knowledge…Continue
Added by Annie Kennedy on September 14, 2016 at 11:30am — No Comments
With the promise of drug approvals on the horizon, access to these therapies will be critical to our Duchenne community. PPMD has been leading work related to Duchenne and rare disease community access issues on multiple fronts.
The Institute for Clinical and…Continue
Added by Annie Kennedy on September 14, 2016 at 11:25am — No Comments
The global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This year a theme was chosen that is not only critical to changing the diagnostic odyssey for people with…Continue
Added by Annie Kennedy on September 1, 2016 at 1:30pm — No Comments
I have a confession…
I have had the privilege of serving as an advocate for families whose children have Duchenne within school systems for more than 20 years, have attended hundreds of IEPs and 504 Plan meetings, and have given even more school presentations, assemblies and in-services about Duchenne… but this year is completely different.…Continue
Added by Annie Kennedy on August 29, 2016 at 1:30pm — No Comments
On Friday, PPMD submitted additional comments to the latest version of PDUFA VI. At this point, there are few members of our Duchenne community who haven’t heard the acronyms ‘PDUFA’ or ‘FDASIA’. But, it’s possible that there are many among us who aren’t aware just how…Continue
Added by Annie Kennedy on August 19, 2016 at 1:38pm — No Comments
We know that many within our community have long been advocating for and following the passage of The Achieving a Better Life Experience (ABLE) Act and subsequent IRS rules and state implementation. In June, the first state ABLE programs launched with the initiation of Ohio’s STABLE program. At …
Added by Annie Kennedy on August 12, 2016 at 9:52am — No Comments
Our Duchenne community’s life-saving research efforts are funded through a number of public and private sources. Beginning in FY 2011, the federal funding to Duchenne began to include the Congressionally Directed Medical Research Program (CDMRP) through the Department of Defense. Between…Continue
Added by Annie Kennedy on June 16, 2016 at 11:00am — No Comments
As you know, PPMD has been helping to lead national advocacy efforts around the 21st Century Cures/ Senate Innovation legislation for more than two years and we now have a very short window of opportunity in which the Senate can act to finalize this legislation and then both chambers can…Continue
Added by Annie Kennedy on June 7, 2016 at 1:00pm — No Comments
It's been a rough few weeks for this community, but our efforts to shape and reshape access barrier issues for our community continue -- and last Wednesday we took a giant leap forward. It was a pivotal day that I was proud to be a part of!
The federal intra-agency Muscular Dystrophy Coordinating Committee (MDCC) held its…Continue
On Saturday, Duchenne community members from six states gathered together at the beautiful facilities at Kennedy Krieger in Baltimore – one of PPMD’s Certified Duchenne Care Centers – to hear the latest developments on clinical trials, clinical care practices,…Continue
Added by Annie Kennedy on April 18, 2016 at 12:30pm — No Comments
Our Duchenne community is on our way to achieving ANOTHER milestone.
MD-CARE Act passed 2001
MD-CARE Act reauthorized 2008
MD-CARE Act Amended 2014
Heavy Engagement within PDUFA V; FDASIA reauthorization 2012
Today, March 9, 2016 on our way to another banner day with the…Continue
Added by Annie Kennedy on March 9, 2016 at 11:30am — No Comments
For nearly two years, PPMD has been working with Congressional champions and industry partners to help lead an effort to streamline the regulatory pathways and review processes for targeted therapeutics (which would include such things as ‘follow on exons’) by clarifying the FDA’s existing authority to leverage data previously used in the approval of a targeted product when approving a new therapy. These efforts have yielded a provision in the House of Representatives…Continue
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…Continue
Added by Annie Kennedy on January 13, 2016 at 1:58pm — No Comments