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March 2017 Blog Posts (12)

PPMD’s #EverySingleOne Tour: Seattle, WA

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…


Added by PPMD on March 31, 2017 at 11:30am — No Comments

Summit to Extend Ongoing PhaseOut DMD Clinical Trial of Ezutromid in Patients with Duchenne Muscular Dystrophy

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…


Added by PPMD on March 27, 2017 at 11:25am — No Comments

Understanding Health Insurance Options: A Guide for People Living with Duchenne Muscular Dystrophy and their Caregivers

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:

  • Private Health Insurance: Families and individuals obtain…

Added by PPMD on March 27, 2017 at 10:30am — 1 Comment

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

ENMC Workshop Report: Airway Clearance Techniques in Neuromuscular Disorders

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…


Added by Kathi Kinnett on March 17, 2017 at 4:00pm — 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

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