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PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 2: Optimizing Clinical Trials

This week, PPMD convened Duchenne industry partners, experts in adaptive trial design and biostatistics, and innovative partners from outside of the Duchenne world who’ve revolutionized their clinical trial spaces for the second meeting of our Duchenne Drug Development Roundtable's 3-part meeting series, focusing on Clinical…


Added by Abby Bronson on March 3, 2017 at 10:00am — No Comments

PPMD Joins Rare Disease Advocates in Letter to President

PPMD joined 200 patient organizations in a letter to President…


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…


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

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…


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

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…


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

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 …


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

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


Today, Marathon…


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

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 (…


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

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.…


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

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…


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

PPMD Awards $600,000 Grant to NJIT & Talem for Next Phase of Upper Extremity Exoskeleton



Ensuring that the worlds of teens and adults with Duchenne 'grow bigger', as they grow older by expanding their reach – both literally and figuratively.


Combing your hair. Lifting a glass to your mouth. Washing your hands. Pressing elevator…


Added by PPMD on January 18, 2017 at 9:00am — 1 Comment

Meet the 2017 PPMD Adult Advisory Committee (PAAC)

Years ago, a friend of mine named Tom Bailey who had Duchenne wrote a memoir that he entitled, Moving Mountains Without Muscles. As I look at the tremendous accomplishments of PPMD’s Adult Advisory Committee since we first launched this leadership group 2 years ago, I am astounded by how…


Added by Annie Kennedy on January 16, 2017 at 7:30pm — No Comments

ATTENTION: All Patients on Masters’ Deflazacort

As you know, the FDA is currently reviewing Marathon Pharmaceutical’s New Drug Application (NDA) for deflazacort for the treatment of Duchenne. It…


Added by PPMD on January 16, 2017 at 6:30pm — No Comments

New Resource to Help Navigate EXONDYS 51 Access

Prior to the approval of EXONDYS 51™, we knew that access to emerging therapies would be a process – and an often challenging process at…


Added by Annie Kennedy on January 13, 2017 at 11:00am — No Comments

New Research & Clinical Trial Tools on PPMD's DuchenneConnect!

We are excited to announce three new features now available on PPMD's DuchenneConnect! These tools are meant to help you in your journey as you gather information around potential research studies and potential new exon…


Added by Ann Lucas on January 12, 2017 at 1:24pm — No Comments

Akashi Therapeutics Provides Update on Three Duchenne Compounds

This weekend, Akashi Therapeutics announced they are working toward having three novel, complementary compounds in the clinic in 2017 with potential to treat all Duchenne patients independent of their specific genetic…


Added by PPMD on January 11, 2017 at 9:00am — No Comments

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