The PPMD Every Single [One] Tour had an amazing turnout last weekend in Seattle, WA! Nearly 100 parents, patients and professionals gathered at Seattle Children’s Hospital to talk and learn about research, care, therapy, and clinical…Continue
Added by PPMD on March 31, 2017 at 11:30am — No Comments
Summit Therapeutics announced today that it will proceed with the planned extension phase of PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid, subject to regulatory approval.
This follows an interim review of the safety and tolerability data from the ongoing trial by an independent Data…Continue
Added by PPMD on March 27, 2017 at 11:25am — No Comments
Health insurance is a contract between you and an insurance company. Health insurance helps pay your medical bills when you need care.
There are two major types of health insurance:
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
The European Neuromuscular Center (ENMC) is a global organization that gathers expertise from around the world to address many issues and concerns of, and advance research and treatments for, neuromuscular…Continue
Added by Kathi Kinnett on March 17, 2017 at 4:00pm — 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