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ReveraGen BioPharma Receives FDA Fast Track Designation for Vamorolone

Today ReveraGen BioPharma Inc, announced that the FDA has granted Fast Track designation for vamorolone (VBP15) for the treatment of Duchenne.  This designation can speed the review of efficacy and safety data for vamorolone in people with Duchenne, potentially leading to more rapid regulatory approval.…


Added by PPMD on March 24, 2017 at 12:30pm — No Comments

Community Update from PTC

Today, PTC Therapeutics provided the following update to the Duchenne community regarding their acquisition of Emflaza™ (deflazacort) from Marathon Pharmaceuticals. We appreciate PTC keeping our community updated during this transition.

March 24, 2017

Dear Duchenne…


Added by PPMD on March 24, 2017 at 10:38am — No Comments

At Long Last, PDUFA VI Hearings Have Begun – But There’s a Twist

Wow...what a week in Washington! President Trump released his budget proposal, the American Health Care Act moves to the floor of the House for a vote today, the U.S. Supreme Court ruled on a landmark case around the rights of students with disabilities within the public school system, and at long last after months of our Duchenne…


Added by Annie Kennedy on March 23, 2017 at 10:30am — No Comments

Akashi Therapeutics Receives FDA Clearance to Resume HT-100 Clinical Development

PPMD is pleased to learn that the FDA has completed its review and concluded that Akashi Therapeutics may resume clinical development of HT-100 (delayed-release halofuginone) in people with Duchenne (click here to read Akashi's press release). Preventing fibrosis is an important target and an essential piece of a combination of therapies that will be…


Added by PPMD on March 22, 2017 at 9:00am — No Comments

ACTION ALERT: Make Your Voice Heard on New Healthcare Proposal

“Who’s on first?”

Remember that classic Abbott & Costello routine? Well, trying to track the quickly-shifting healthcare bill has felt much like watching that routine. Complex, confusing, and exhausting. But the stakes here are no laughing matter.




Added by Annie Kennedy on March 21, 2017 at 10:00am — No Comments

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

PAAC Leadership Conference 2017

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

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