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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 …

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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…

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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…

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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…

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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 …

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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…

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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…

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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,…

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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 …

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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…

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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…

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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…

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

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

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

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

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

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

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

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

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