Earlier this month, I testified on behalf of PPMD and the national Duchenne community before the Advisory Committee on Heritable Disorders in Newborns and Children – the U.S. federal advisory committee on newborn…

Call & Email Your Senators Today!

Both the House & Senate have now released their Tax Reform proposals, each with variations of similar policy impacts and changes that we feel an obligation to alert you to. It is anticipated that both Chambers will move this legislation swiftly and work to complete their votes during the week…

Progress! Today, Anthem issued a revised policy regarding Sarepta’s EXONDYS 51. In the revision, Anthem declared EXONDYS 51 medically necessary for the treatment of Duchenne muscular dystrophy when all of the following criteria are met:

1. Individual has a confirmed diagnosis of Duchenne muscular dystrophy; and
2. Individual must be ambulatory (with or without needing an…

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?

Hear first-hand what it's like to serve on the PPMD Adult Advisory Committee (PAAC) from current members…

Call & Email Your Senators Today!

Yesterday, the Senate released the Tax Cuts and Jobs Act which includes many issues that we feel an obligation to alert you to.

While PPMD does not typically engage in legislation related to tax reform, this proposal (being referred to as…

On October 25, PTC Therapeutics shared the news that the FDA had issued a Complete Response Letter regarding their application for ataluren, noting that FDA had stated that “an additional adequate and well-controlled…