Great news – The ABLE program enhancements have been re-introduced in Congress and are gaining support. This package of bills include improvements to the original ABLE program that directly impact our community, here’s how:
On April 4th, a bi-partisan group of Members of Congress,…Continue
Added by Annie Kennedy on April 14, 2017 at 9:00pm — No Comments
In late March, PPMD joined 87 organizations to issue a letter calling on Congress to take a hard look at the likely significant and…Continue
Added by Annie Kennedy on April 13, 2017 at 9:30pm — No Comments
PPMD is proud to sign on to letters sent both to the Senate and the House asking that they provide at least $29.8 million to the Centers for Disease Control and Prevention’s (CDC) Newborn Screening Quality Assurance Program (NSQAP) and at least $19.9 million to the Health Resources and Services…Continue
Added by Annie Kennedy on April 12, 2017 at 9:00pm — No Comments
Iowa Children’s Hospital marked our third stop on PPMD’s 2017 Every Single One Tour, held on April 1, 2017. The amazing staff at Iowa Children’s Hospital helped make this a wonderful day and we can see…Continue
Added by PPMD on April 7, 2017 at 1:30pm — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named UC Davis Department of Physical Medicine and Rehabilitation Neuromuscular Disease Clinic (UC…Continue
Added by PPMD on April 6, 2017 at 12:30pm — No Comments
Accelerating trials. Protecting our robust therapeutic pipeline. Ensure as many hits on goal as we can.
Priorities shared by all of us.
And to achieve this, PPMD has convened our industry partners through our Duchenne Drug Development Roundtable…Continue
Added by Annie Kennedy on April 5, 2017 at 11:00am — No Comments
Later this spring – likely on or about May 18 – FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s Essence trial.
Once again, our community is at a pivotal moment in our history where we…Continue
Added by Annie Kennedy on April 5, 2017 at 10:00am — No Comments
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:
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
Added by Annie Kennedy on February 27, 2017 at 7:30pm — 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