PPMD's Blog (474)

83 International Experts Submit Letter to FDA Encouraging Approval of Ataluren

PPMD applauds the group of 83 international experts with experience caring for Duchenne patients around the globe who have written a letter to the FDA encouraging approval of ataluren. With hundreds of patients accessing ataluren through the…

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Added by PPMD on October 9, 2017 at 10:36am — No Comments

News from 2017 World Muscle Society: Part 1

 

Annie Kennedy and Kathi Kinnett from the PPMD team are attending the 22nd International Annual Congress of the World Muscle Society and will be reporting back over the next couple of days. More…

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Added by PPMD on October 6, 2017 at 1:30pm — No Comments

Catabasis Reports Positive Results from Open-Label Extension of MoveDMD Trial with Edasalonexent

Catabasis Pharmaceuticals, Inc. today reported new positive efficacy results following 24 and 36 weeks of treatment with 100 mg/kg/day oral edasalonexent in the MoveDMD Phase 2 trial open-label…

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Added by PPMD on October 4, 2017 at 10:30am — 1 Comment

Letter from PTC Therapeutics to the Community Following FDA Ad Comm

PTC Therapeutics President and CEO, Stuart Peltz, Ph.D has shared a letter with the Duchenne community following last week's Advisory Committee Meeting at…

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Added by PPMD on October 3, 2017 at 9:30am — No Comments

September 28: FDA Advisory Committee Meeting for Ataluren - Live Webcast Information

On Thursday, September 28, an FDA Advisory Committee will review ataluren for potential approval and use within our Duchenne community.



The Duchenne community is joining forces in a show of strength to…

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Added by PPMD on September 27, 2017 at 6:42pm — No Comments

PPMD Continues Grant Supporting Clinic Liaisons for Certified Duchenne Care Center Program

PPMD is excited to announce that we will continue a grant program to help support our Certified Duchenne Care Center (CDCC) Program. The CDCC Program is a network of…

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Added by PPMD on September 27, 2017 at 12:30pm — No Comments

Are You Ready for Coach To Cure MD This Saturday?

This weekend, nearly every televised College Football game around the country will have coaches wearing Coach To Cure MD armbands, tweeting about Duchenne, and encouraging fans to text a…

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Added by PPMD on September 26, 2017 at 1:00pm — No Comments

Briefing Documents for Ataluren Ad Comm Released

The briefing documents for the September 28…

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Added by PPMD on September 26, 2017 at 9:30am — No Comments

Santhera Receives Negative Opinion from the CHMP for Raxone® and Intends to Appeal this Opinion

Santhera Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion for its Type II extension application for Raxone® (idebenone) in Duchenne. Santhera plans to appeal the opinion and will seek a re-examination by the CHMP. We appreciate…

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Added by PPMD on September 15, 2017 at 8:00am — No Comments

PPMD Designates Nemours/Alfred I duPont Hospital for Children a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Nemours/Alfred I duPont Hospital for Children (Nemours) in Wilmington, Delaware our 17th Certified Duchenne Care Center.

PPMD’s Certified Duchenne Care Center Program, which supports standardized, comprehensive care and services for all people…

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Added by PPMD on September 14, 2017 at 8:30pm — No Comments

Reminder: Deadline to Submit Written Comments for Ataluren Ad Comm is Thursday, September 14

The deadline to submit a written comment for the FDA Advisory Committee Meeting on ataluren is this upcoming Thursday, September 14th.

 

To make a comment,…

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Added by PPMD on September 12, 2017 at 9:30am — No Comments

The 2018 End Duchenne Tour – Coming to a City Near You!

PPMD is excited to announce next year’s lineup of areas we will visit as part of our End Duchenne Tour!



Combining each of the pillars that make up PPMD’s mission, the End Duchenne Tour brings updates on research, advocacy, and care to cities across the country, featuring a roster of leading experts in the Duchenne…

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Added by PPMD on September 8, 2017 at 5:30pm — No Comments

Sarepta Therapeutics Announces Positive Results from 4053-101 Study

Sarepta Therapeutics, Inc. today announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen…
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Added by PPMD on September 6, 2017 at 10:00am — No Comments

Roche Provides Community Update on BMS-986089 (RG6206)

Earlier this year, Bristol-Myers Squibb (BMS) announced it had entered into an agreement to license BMS-986089 (RG6206), an anti-myostatin adnectin in development for Duchenne muscular dystrophy, to Roche. Today, the Roche team has provided an update to our…

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Added by PPMD on September 5, 2017 at 9:30am — No Comments

How You Can Participate in the Ataluren Ad Comm

The Duchenne community is now preparing for the September 28 Advisory Committee Meeting for PTC’s therapy ataluren. Whether submitting written testimony and/or attending the Ad Comm in person, there are multiple…

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Added by PPMD on August 22, 2017 at 11:00am — No Comments

PPMD Designates Gillette Children’s Specialty Healthcare a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Gillette Children's Specialty Healthcare Neuromuscular Clinic (Gillette) our 16th Certified Duchenne…

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Added by PPMD on July 13, 2017 at 11:21am — No Comments

PPMD to Host Duchenne Drug Development Roundtable During 23rd Annual Connect Conference

 
PPMD will host our annual Duchenne Drug Development Roundtable…
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Added by PPMD on June 30, 2017 at 1:49pm — No Comments

PPMD Convenes International Experts for Inflammation and Immunity in Duchenne Muscular Dystrophy Workshop

Today, PPMD is convening medical experts from around the world for a workshop exploring inflammation and immunity in Duchenne on the eve of our 23rd Annual Connect Conference.



In January, PPMD organized a Critical Path Innovation Meeting with the FDA focused on…

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Added by PPMD on June 28, 2017 at 2:19pm — No Comments

FDA Hiring Freeze Lifted

Recently, FDA Commissioner Scott Gottlieb announced that the hiring freeze, instituted on January 23, has been lifted.

This reversal means that the FDA can begin to fill vacancies – needed resources for continued research into rare disease treatments and for the review and approval of new medicines.

PPMD is proud to have been one…

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Added by PPMD on June 15, 2017 at 11:53am — No Comments

PPMD Signs On to DoD Funding Letters

Once again, PPMD has signed on to letters to leaders in both the House and Senate, requesting they continue to support the critical and highly successful defense health research programs funded through the Congressionally Directed Medical Research Programs (CDMRP) at the Department of Defense (DoD) for FY 2018.…

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Added by PPMD on June 15, 2017 at 11:30am — No Comments

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