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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


Staff
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…

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


Staff
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…

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


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


Staff
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.…

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


Staff
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…

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Added by PPMD on March 24, 2017 at 10:38am — No Comments


Staff
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…

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Added by Annie Kennedy on March 23, 2017 at 10:30am — No Comments


Staff
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…

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Added by PPMD on March 22, 2017 at 9:00am — No Comments


Staff
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.

 

We…

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Added by Annie Kennedy on March 21, 2017 at 10:00am — No Comments


Staff
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…

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Added by Kathi Kinnett on March 17, 2017 at 4:00pm — No Comments


Staff
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…

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Added by Abby Bronson on March 3, 2017 at 10:00am — No Comments


Staff
PPMD Joins Rare Disease Advocates in Letter to President

PPMD joined 200 patient organizations in a letter to President…

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Added by Annie Kennedy on February 27, 2017 at 7:30pm — No Comments

Recap: PPMD's Adult Advisory Committee (PAAC) Leadership Meeting and 2017 Advocacy Conference

Heartening. That’s how I found last week’s PPMD PAAC Leadership and Advocacy Conference.  Every time the whole community gets together, I leave feeling EMPOWERED.  I like that word. Good word isn’t it? There is no limit to what I can do for my community. And I imagine I’m not the…

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Added by Buddy Cassidy on February 27, 2017 at 4:00pm — No Comments


Staff
Duchenne Research Update: February 2017

 

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…

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Added by Abby Bronson on February 24, 2017 at 1:29pm — No Comments


Staff
PPMD Designates Ann & Robert H. Lurie Children's Hospital of Chicago a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Ann & Robert H. Lurie Children’s…

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Added by PPMD on February 16, 2017 at 12:00pm — No Comments

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