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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

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

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

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

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

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

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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