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.…Continue
Added by PPMD on March 24, 2017 at 12:30pm — No Comments
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
Added by PPMD on March 24, 2017 at 10:38am — No Comments
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…Continue
Added by Annie Kennedy on March 23, 2017 at 10:30am — No Comments
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…Continue
Added by PPMD on March 22, 2017 at 9:00am — No Comments
“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
Today we learned that PTC Therapeutics has entered into an asset purchase agreement with Marathon Pharmaceuticals, LLC …Continue
Added by PPMD on March 16, 2017 at 11:00am — No Comments
Added by PPMD on March 6, 2017 at 9:00am — No Comments
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…Continue
Added by Abby Bronson on March 3, 2017 at 10:00am — No Comments
Added by Annie Kennedy on February 27, 2017 at 7:30pm — No Comments
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…Continue
Added by Buddy Cassidy on February 27, 2017 at 4:00pm — No Comments
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…Continue
Added by Abby Bronson on February 24, 2017 at 1:29pm — No Comments
Added by PPMD on February 16, 2017 at 12:00pm — No Comments
In light of the pause in commercialization of EMFLAZA™ (deflazacort)…Continue
Added by PPMD on February 15, 2017 at 1:30pm — No Comments
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 …Continue
Added by PPMD on February 15, 2017 at 10:30am — No Comments
UPDATE: February 13, 2017, 5:10 PM -- An additional statement from Marathon has been added to the blog
Added by PPMD on February 13, 2017 at 4:00pm — No Comments
UPDATE 2/10: Marathon has released links to new resources, including the EMFLAZA website (https://emflaza.com) and a Patient Support FAQ document (…Continue
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
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…Continue
Added by Abby Bronson on February 1, 2017 at 10:00am — No Comments
Added by PPMD on January 31, 2017 at 4:08pm — No Comments
Added by Pat Furlong on January 30, 2017 at 10:00am — No Comments