PPMD Leads Duchenne Advocates to Capitol Hill to Accelerate Therapies and Quality Care

PPMD's Annual Advocacy Conference Focused on Implementing MD-CARE Act Amendments & Strengthening Patient Voice at FDA

Just months after helping achieve enactment of amendments to the landmark Muscular Dystrophy CARE Act (MD-CARE Act) law, Parent Project Muscular Dystrophy (PPMD) advocates are returning to Capitol Hill for the organization's Annual Advocacy Conference, a February staple in the Duchenne muscular dystrophy community.

More than 150 advocates – people with Duchenne, parents, siblings, grandparents, and other family members and friends will be meeting with more than 200 House and Senate offices today and tomorrow to continue advancing federal policies to accelerate the development and delivery of Duchenne therapies and to ultimately end Duchenne.

This year's advocacy conference weekend began with the inaugural meeting of PPMD's Adult Advisory Committee on Saturday, with several of the panel members participating as advocates later this week.

"The fact that we now have a thriving adult advisory committee is a testament to how far we have come in the more than 20 years since PPMD was founded. I am so deeply inspired by this group of young men and all that they have accomplished during their lives, and am honored that they will lend their voices to our advocacy effort."

-- PPMD Founding President Pat Furlong

This year's PPMD advocacy agenda focuses on implementing many of the provisions contained in the MD-CARE Act Amendments (Public Law 113-166), including expansion of the research agenda, updating of existing care standards that have done so much to lengthen and enhance patient life, and strengthening programs focused on assisting adults with the Duchenne.

Advocates will also support an expansion of a promising Duchenne newborn screening pilot program and will continue pushing for an expanded patient voice in the Food and Drug Administration drug review process.

Last year, PPMD submitted to FDA the first-ever patient-initiated guidance document to help reduce the time and cost associated with Duchenne drug development. Nearly eight months later, that document is still pending with FDA, and advocates will be urging that the agency move it forward.

"We must do everything we can to ensure that the day an application for a Duchenne therapy arrives at the FDA, the agency moves as quickly as possible to review the submission and takes into account the invaluable patient and caregiver perspectives and preferences when making such critical decisions."

-- PPMD Founding President Pat Furlong

In addition to meeting with Members of Congress and staff, PPMD will host a Congressional briefing focused on the work the organization has been leading to enhance the role of the patient in the drug review process.

To learn more about PPMD's Annual Advocacy Conference and our extensive advocacy agenda, please visit our website.

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Comment by Shelly on February 23, 2015 at 7:31pm
We are always with PPMD. A great job to save many lives. BEST OF LUCK PPMDers!!!!!
Comment by David on February 23, 2015 at 12:08pm
Politicians, more than anything else, want a record of success. That is the DMD story, we are winning. The band wagon is big enough for them all! Best of luck PPMDers

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