PPMD is continuously seeking innovative opportunities to impact the process of developing therapies for Duchenne. Opportunity comes in many forms - funding promising research, advancing care standards for all patients, and advocating for key changes in federal policy.
Earlier this year Congress launched the 21st Century Cures Initiative, a comprehensive look at what can be done to accelerate the pace of cures in American therapeutic…Continue
Added by Ryan Fischer on September 15, 2014 at 12:00pm — No Comments
Timing is everything. When I think back on how I found Pat, Kimberly, and PPMD ten years ago to the month, I often think about the events that led to that faithful interview in the fall of September 2004. Sure, maybe some of it was luck, but I also believe there was a higher power at work. When I spoke to Pat the first time…Continue
Added by Ryan Fischer on September 10, 2014 at 4:00pm — No Comments
Congress has returned from their Summer recess and it is time to re-engage them on the MD-CARE Act Amendments. We need each member of our community to reach out to your Senators and urge them to tell leadership to bring the…Continue
Added by Ryan Fischer on September 9, 2014 at 10:35am — No Comments
Sure, we’re in the lazy days of summer and Congress is on recess, but now is not the time to slow down on the MD-CARE Act. Let’s keep our momentum going. Take a minute to thank the House for passing the bill and tell the Senate why we need them to take up the…Continue
Added by Ryan Fischer on August 13, 2014 at 9:30am — No Comments
MD-CARE Act Clears Senate HELP Committee
Today, the Senate HELP Committee approved the MD-CARE Act - another huge step in the right direction for our bill. This…Continue
Added by Ryan Fischer on July 23, 2014 at 1:00pm — No Comments
Please take 1 minute to email your Senators!
Today, a key Senate committee will …Continue
Added by Ryan Fischer on July 23, 2014 at 11:00am — No Comments
A group of Seattle-based muscular dystrophy researchers has been awarded a prestigious $6.3 million grant from the National Institute of Health (NIH) to establish a Senator Paul D. Wellstone Cooperative Research Center. Congratulations to Dr. Chamberlain and…
Added by Ryan Fischer on July 18, 2014 at 11:28am — No Comments
Please take 1 minute to email your Senators!
Yesterday, a key Senate committee announced that it will …Continue
Added by Ryan Fischer on July 17, 2014 at 12:30pm — No Comments
Progress! Today the House Energy & Commerce committee voted unanimously to advance H.R. 594, the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2014. This action moves the bill to consideration and hopefully passage by the full…Continue
Added by Ryan Fischer on July 15, 2014 at 10:30am — No Comments
Progress! Yesterday the House Energy & Commerce Subcommittee on Health voted via unanimous voice vote to advance H.R. 594, the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2014. This action moves the bill one step…Continue
Added by Ryan Fischer on June 18, 2014 at 9:30am — No Comments
Strength Happens Together
For the past four months, over 80 experts in Duchenne (including patient representatives and groups, industry, clinicians, and researchers) have been working…Continue
Added by Ryan Fischer on June 4, 2014 at 4:00pm — No Comments
On May 20th, the House Energy and Commerce Subcommittee on Health held a hearing entitled, 21st Century Cures: The President’s Council of Advisors on Science and Technology (PCAST) Report on Drug Innovation.
The hearing was led by Chairman Joe Pitts (R-PA) and is part of the new 21st Century Cures initiative that seeks to accelerate the discovery, development and delivery of promising new treatments to patients.…Continue
Added by Ryan Fischer on May 22, 2014 at 3:30pm — No Comments
The process for writing a Draft Guidance on Duchenne for the FDA and industry is in full swing.
As you know, the purpose of this guidance is to assist sponsors in the clinical development of medical products (i.e., human drugs and…Continue
Added by Ryan Fischer on May 9, 2014 at 9:30am — No Comments
Originally signed into law in 2001, the MD-CARE Act has birthed key discoveries in the muscular dystrophies, including the scientific justification for why exon skipping therapies could potentially work in humans.
Without it being…Continue
Added by Ryan Fischer on May 8, 2014 at 4:30pm — No Comments
Thanks to your efforts, we closed the House and Senate funding letters with the support of 30 House members and 12 Senators. Great work! We appreciate everyone who sent emails and made calls to their members of Congress.
The members below collectively sent a strong message to the House and Senate appropriations committees…Continue
Added by Ryan Fischer on April 15, 2014 at 9:51am — No Comments
As a community, we must continue to demand that the FDA use all available tools given to them by Congress to speed the approval of new therapies to all who so desperately need them. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.
Here are 3 simple actions you can take to support & amplify that message:
Added by Ryan Fischer on March 19, 2014 at 12:10pm — No Comments
Though the MD-CARE Act remains our top priority, we need to ensure that agencies receive adequate funding and direction for the programs that are so critical to our community. One tool we use to ensure that funding and direction is our Congressional funding letter. It has made a tremendous impact on appropriations.
To illustrate this point, let’s look at how our yearly funding letters have impacted the NIH’s…Continue
It's been just one week since the 2014 Advocacy Conference, and we have already upped the number of cosponsors for the MD-CARE Act! We are now at 85 in the House & 21 in the Senate. Please take a moment to reach out to our newest cosponsors listed below thank them.
This community is…Continue
An update on developing "Draft Guidance on the Clinical Investigation of drugs and biologics for Duchenne and Becker muscular dystrophy"
One of PPMD’s priorities is speeding development of reliable diagnostics and safe and effective medications for all individuals diagnosed with Duchenne or Becker…Continue
Added by Ryan Fischer on January 28, 2014 at 2:00pm — No Comments
You are invited to join us in Washington (February 23-25) for what is going to be another critical year in Duchenne advocacy. As you know our efforts last year were focused on collecting cosponsors for the MD-CARE Act (MDCA) reauthorization. To date we stand at …Continue
Added by Ryan Fischer on January 16, 2014 at 10:00am — No Comments