PPMD Community

PPMD's Blog (442)

Capricor Reports Significant Improvements in Duchenne Patients Treated with CAP-1002 Investigational Cell Therapy

PPMD is excited to learn that Capricor's investigational cell therapy, CAP-1002, is showing significant improvements in people with Duchenne. We look forward to learning more details about this therapy in a community webinar on Wednesday, November 29 at 1pm eastern. Details coming soon!

Read the release from…

Continue

Added by PPMD on November 15, 2017 at 2:30pm — No Comments

PPMD Submits Community Sign-On Letter to FDA Supporting Approval of Ataluren

PPMD has been continuing to engage with both PTC and the FDA on behalf of the patient community regarding the regulatory review of ataluren.



Earlier this month, …

Continue

Added by PPMD on November 13, 2017 at 1:00pm — No Comments

Wave Life Sciences Initiates Clinical Trial for Lead Program in Duchenne

Wave Life Sciences Ltd. announced the initiation of a global Phase 1 clinical trial for WVE-210201 in Duchenne patients amenable to exon 51 skipping. PPMD is excited by the progress Wave Life Sciences has made in exon skipping. While advances have been made in exon skipping, especially recently, there is more to explore within this technology. We…

Continue

Added by PPMD on November 6, 2017 at 9:09am — No Comments

Sarepta & Nationwide Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne

More good news in gene therapy! Today, Sarepta Therapeutics announced that Nationwide Children’s Hospital has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for the micro-dystrophin gene therapy program. The Phase 1/2a clinical trial, now open to enrollment and scheduled for initiation by mid-November, is designed to assess the safety and tolerability of AAVrh74.MHCK7.micro-Dystrophin in individuals…

Continue

Added by PPMD on November 6, 2017 at 8:30am — No Comments

Sarepta Therapeutics & Nationwide Children’s Hospital Announce FDA Clearance of the IND Application for the GALGT2 Gene Therapy Program

Once again, PPMD is excited to see industry, patient groups, and research partnering, as today Nationwide Children's Hospital and Sarepta Therapeutics announced the achievement of an important milestone in…

Continue

Added by PPMD on November 3, 2017 at 8:39am — No Comments

Letter from PTC Therapeutics to the Community Following FDA Decision

PTC has shared a letter with the Duchenne community in regards to the recent FDA decision. PTC will be holding a community call on Friday, October 27 at 12:00 PM EST to discuss next steps with the community. Please e-mail your…

Continue

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

News from 2017 World Muscle Society: Part 2

Annie Kennedy and Kathi Kinnett from the PPMD team recently attended the 22nd International Annual Congress of the World Muscle…

Continue

Added by PPMD on October 9, 2017 at 12:30pm — No Comments

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…

Continue

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…

Continue

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…

Continue

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…

Continue

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.…

Continue

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…

Continue

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…

Continue

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

Briefing Documents for Ataluren Ad Comm Released

The briefing documents for the …

Continue

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…

Continue

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,…

Continue

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…

Continue

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…

Continue

Added by PPMD on September 8, 2017 at 5:36pm — No Comments

Monthly Archives

2017

2016

2015

2014

2013

2012

2011

2010

© 2017   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service