Great news – The ABLE program enhancements have been re-introduced in Congress and are gaining support. This package of bills include improvements to the original ABLE program that directly impact our community, here’s how:

On April 4th, a bi-partisan group of Members of Congress, including Senators Richard Burr (R-NC), Bob Casey (D-PA), Jerry…

In late March, PPMD joined 87 organizations to issue a letter calling on Congress to take a hard look at the likely significant and life-threatening consequences of the American Health Care Act on millions of…

PPMD is proud to sign on to letters sent both to the Senate and the House asking that they provide at least $29.8 million to the Centers for Disease Control and Prevention’s (CDC) Newborn Screening Quality Assurance Program (NSQAP) and at least $19.9 million to the Health Resources and Services Administration’s (HRSA) Heritable Disorders…

Accelerating trials. Protecting our robust therapeutic pipeline. Ensure as many hits on goal as we can.

Priorities shared by all of us.

And to achieve this, PPMD has convened our industry partners through our Duchenne Drug Development Roundtable and held a series of small, targeted meetings this winter, each focused…

Later this spring – likely on or about May 18 – FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s Essence trial.

Once again, our community is at a pivotal moment in our history where we can affect change and improve the clinical trial experience for our…