Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. The community has anxiously awaited an update on both access and pricing ever since the deal between PTC and Marathon was announced.
On today’s call, we…Continue
Added by PPMD on May 8, 2017 at 5:38pm — No Comments
PTC Therapeutics has released a letter to the community and will host a conference call for the Duchenne community to discuss the launch plans for EMFLAZA™ (deflazacort) on Monday, May 8, 2017 at 3:00 PM EDT (call-in details are below).
We may have lost the battle — but the war is far from over.
Some days are hard to make sense of. Today feels like one of those for many as we watched the House votes tally this afternoon.
This community knows better than most that our healthcare system needs repair and innovation. But the proposals that have been brought forward have not represented enhancements to healthcare…
Added by Annie Kennedy on May 4, 2017 at 6:00pm — No Comments
The newest version of the GOP HealthCare Bill is coming to a vote by 1:30 PM EDT this afternoon!Continue
Added by Ryan Fischer on May 4, 2017 at 12:00pm — No Comments
The current version of American Health Care Act (AHCA) directly threatens essential health benefits (EHBs), Medicaid eligibility and funding, and vital health care protections for our Duchenne community.
I wish there was a way to sugar coat this. Find the silver lining. See the glass as…
Added by Annie Kennedy on April 28, 2017 at 9:30am — No Comments
PTC Therapeutics, Inc. will host a conference call for the Duchenne community to discuss the launch plans for Emflaza™ (deflazacort) on Monday, May 8, 2017 at 3:00pm (ET).
The call can be…Continue
Added by PPMD on April 26, 2017 at 10:00am — No Comments
Capricor Therapeutics today announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne.
We are excited to see these results and to see the field of cell…Continue
On May 18, 2017, FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s…Continue
Added by PPMD on April 24, 2017 at 12:30pm — No Comments
Today, PTC Therapeutics announced that they have completed their acquisition of Emflaza™ (deflazacort) ahead of schedule, following early conclusion of the anti-trust review.
PTC will be hosting a call with the Duchenne community the week of May 8 to provide details on the plan for the launch of Emflaza. We look forward to learning more from PTC in the…Continue
Added by PPMD on April 20, 2017 at 4:00pm — No Comments
The Duchenne community has waited 20 years to be where we are. We are reminded daily of how far we have come, how the landscape has changed, how there are close to 20 clinical trials to choose from. While these changes within are landscape bring us to a hopeful time in Duchenne, the journey to this point has not been painless for the families…Continue
Added by Kathi Kinnett on April 20, 2017 at 12:30pm — No Comments
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, including Senators Richard Burr (R-NC), Bob Casey (D-PA), Jerry…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 life-threatening consequences of the American Health Care Act on millions of…Continue
Added by Annie Kennedy on April 13, 2017 at 9:30pm — No Comments
Bristol-Myers Squibb (BMS) today announced that it has entered into an agreement to license BMS-986089, an anti-myostatin adnectin in development for Duchenne muscular dystrophy, to Roche. …Continue
Added by PPMD on April 13, 2017 at 10:03am — 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 Administration’s (HRSA) Heritable Disorders…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 why they are one of the 15 …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 Davis) our 15th …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 and held a series of small, targeted meetings this winter, each focused…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 can affect change and improve the clinical trial experience for our…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