PPMD joined 200 patient organizations in a letter to President Trump about the challenges facing rare disease patients in America and the vital role that federal agencies play in helping to accelerate the…

Heartening. That’s how I found last week’s PPMD PAAC Leadership and Advocacy Conference.  Every time the whole community gets together, I leave feeling EMPOWERED.  I like that word. Good word isn’t it? There is no limit to what I can do for my community. And I imagine I’m not the only one who feels like this. Or at least I hope you feel empowered too.  And if…

An Overview of Your Impact in Action

PPMD Grant Awards in the last Quarter of 2016 and first part of 2017.

PPMD Launches Long-Term…

In light of the pause in commercialization of EMFLAZA™ (deflazacort) by Marathon…

Children’s Hospital Los Angeles (CHLA) marked our first stop of the New Year on PPMD’s 2017 Every Single One Tour, held on January 21, 2017. We were thrilled to not only be holding another tour stop at one of …

UPDATE: February 13, 2017, 5:10 PM -- An additional statement from Marathon has been added to the blog

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Today, Marathon Pharmaceuticals announced that they will pause the commercialization of…

UPDATE 2/10: Marathon has released links to new resources, including the EMFLAZA website (https://emflaza.com) and a Patient Support FAQ document (…

Following decades of strategic advocacy, research investment, and biopharmaceutical development, the U.S. Duchenne community recently arrived in the post-approval space and is poised to receive additional novel therapies from a robust innovation pipeline.

Parent Project Muscular Dystrophy (PPMD) is deeply…

Better. Faster. Now.

How do we apply the ‘regulatory learnings’ of the past 18 months back into our robust Duchenne pipeline to ensure as many effective therapies yield from our pipeline as possible?

Under the leadership of PPMD's Duchenne Drug Development Roundtable (DDDR), our community is determined to figure that out. Today is the first of a…