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April 2017 Blog Posts (14)


Staff
ACTION ALERT: ­Immediate Action Needed – Revised AHCA Bill Threatens Duchenne Community’s Access to Health Care Coverage

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…

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Added by Annie Kennedy on April 28, 2017 at 9:30am — No Comments


Staff
PTC Therapeutics Announces Community Conference Call on May 8 to Discuss Launch Plans for Emflaza™ (Deflazacort)

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…

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Added by PPMD on April 26, 2017 at 10:00am — No Comments


Staff
Capricor Therapeutics Announces Positive Six-Month Results from the Randomized Phase I/II HOPE Clinical Trial in Duchenne Muscular Dystrophy

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…

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Added by PPMD on April 25, 2017 at 10:24am — 2 Comments


Staff
FDA Announces Pediatric Advisory Committee Meeting on May 18

On May 18, 2017, FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s…

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Added by PPMD on April 24, 2017 at 12:30pm — No Comments


Staff
PTC Therapeutics Completes Acquisition of Emflaza™ for the Treatment of Duchenne in the U.S.

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…

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Added by PPMD on April 20, 2017 at 4:00pm — No Comments


Staff
PPMD Convenes Meeting to Explore the Burden of Pediatric Clinical Trials

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…

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Added by Kathi Kinnett on April 20, 2017 at 12:30pm — No Comments


Staff
Members of Congress Re-Introduce ABLE Improvement Bills

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,…

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Added by Annie Kennedy on April 14, 2017 at 9:00pm — No Comments


Staff
PPMD Joins Letter to Protect Medicaid

In late March, PPMD joined 87 organizations to issue a letter calling on Congress to take a hard look at the likely significant and…

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Added by Annie Kennedy on April 13, 2017 at 9:30pm — No Comments


Staff
Bristol-Myers Squibb Enters into Agreement with Roche to License Anti-Myostatin Compound

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. …

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Added by PPMD on April 13, 2017 at 10:03am — No Comments


Staff
PPMD Signs Appropriation Letter to Fund Newborn Screening

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…

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Added by Annie Kennedy on April 12, 2017 at 9:00pm — No Comments


Staff
PPMD’s #EverySingleOne Tour: Iowa City, IA

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…

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Added by PPMD on April 7, 2017 at 1:30pm — No Comments


Staff
PPMD Designates UC Davis a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named UC Davis Department of Physical Medicine and Rehabilitation Neuromuscular Disease Clinic (UC…

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Added by PPMD on April 6, 2017 at 12:30pm — No Comments


Staff
PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 3: Clinical Trial Readiness from the Viewpoint of Clinicians and Infrastructure

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…

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Added by Annie Kennedy on April 5, 2017 at 11:00am — No Comments


Staff
PPMD Working with FDA to Plan Landmark Pediatric Advisory Committee Meeting to Improve Clinical Trial Experience

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…

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Added by Annie Kennedy on April 5, 2017 at 10:00am — No Comments

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