February 2017 Blog Posts (10)

Staff PPMD Joins Rare Disease Advocates in Letter to President

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…

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Added by Annie Kennedy on February 27, 2017 at 7:30pm — No Comments

Recap: PPMD's Adult Advisory Committee (PAAC) Leadership Meeting and 2017 Advocacy Conference

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…

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Added by Buddy Cassidy on February 27, 2017 at 4:00pm — No Comments

Staff Duchenne Research Update: February 2017

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…

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Added by Abby Bronson on February 24, 2017 at 1:29pm — No Comments

Staff PPMD Designates Ann & Robert H. Lurie Children's Hospital of Chicago a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Ann & Robert H. Lurie Children’s Hospital of Chicago …

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Added by PPMD on February 16, 2017 at 12:00pm — No Comments

Staff PPMD Recommends Marathon Convene Broad Array of Duchenne Stakeholders to Discuss Re-evaluation of Deflazacort Pricing

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

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Added by PPMD on February 15, 2017 at 1:30pm — No Comments

Staff PPMD’s #EverySingleOne Tour: Los Angeles, CA

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 …

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Added by PPMD on February 15, 2017 at 10:30am — No Comments

Staff Marathon Pharmaceuticals Pauses Commercialization of EMFLAZA™ (deflazacort)

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…

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Added by PPMD on February 13, 2017 at 4:00pm — No Comments

Staff Deflazacort Approved in the U.S. for Duchenne

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

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Added by Pat Furlong on February 9, 2017 at 5:00pm — 2 Comments

Staff PPMD to Host Forum on Access to Emerging Therapies on February 13th, 2017

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.…

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Added by PPMD on February 7, 2017 at 1:57pm — No Comments

Staff PPMD’s Duchenne Drug Development Roundtable: Bringing Industry Together to Accelerate Research

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…

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Added by Abby Bronson on February 1, 2017 at 10:00am — No Comments