PPMD Community

PPMD's Blog (426)

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

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…
Continue

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…

Continue

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…

Continue

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 …

Continue

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…
Continue

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…

Continue

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…

Continue

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…

Continue

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

PPMD’s #EverySingleOne Tour: Gainesville, FL

PPMD's Pat Furlong 

 

Earlier this month, we had the privilege of having our fourth stop in 2017 on …

Continue

Added by PPMD on June 14, 2017 at 10:30am — No Comments

PTC Therapeutics Announces FDA Advisory Committee Meeting for Ataluren

PTC Therapeutics, Inc. today announced that the FDA has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna™). This is another important moment for our community and we look forward to the opportunity to share our collective experiences with Translarna with the FDA. As details on this Ad Comm become…

Continue

Added by PPMD on June 6, 2017 at 8:47am — No Comments

PTC Therapeutics Provides EMFLAZA™ (Deflazacort) FAQ Update

PTC Therapeutics has provided an update regarding EMFLAZA (see below). Please visit EMFLAZA.com for answers to some of the most frequently asked questions PTC has…

Continue

Added by PPMD on June 2, 2017 at 11:30am — No Comments

In Honor of Moms: A Note from Tom Furlong

This Mother’s Day, thank someone for their role in the fight to end Duchenne.

If you’re reading this blog, chances are you know my wife, Pat Furlong. For 23 years now, Pat has been leading Parent Project Muscular Dystrophy (PPMD) and the fight to end Duchenne. Many of you have told me over the years that Pat has been a source of comfort,…

Continue

Added by PPMD on May 10, 2017 at 10:30am — No Comments

PPMD's 2017 Connect Conference Agenda is Now Available!

PPMD is excited to unveil the agenda for our 2017 Connect Conference in Chicago, June 29 - July 2, 2017!

 

We are so proud of the …

Continue

Added by PPMD on May 9, 2017 at 11:00am — No Comments

PPMD's Response to PTC Announcement of EMFLAZA (deflazacort) Pricing & Access

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 to Discuss Launch Plans for Emflaza™ (Deflazacort) during Community Call on Monday, May 8th at 3:00 PM EDT

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).



On a…

Continue

Added by PPMD on May 8, 2017 at 10:00am — 1 Comment

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…

Continue

Added by PPMD on April 26, 2017 at 10:00am — 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