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All Blog Posts (1,634)


Staff
Duchenne Research Update: February 2017

 

An Overview of Your Impact in Action



PPMD Grant Awards in the last Quarter of 2016 and first part of 2017.

 

PPMD Launches Long-Term…

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Added by Abby Bronson on February 24, 2017 at 1:29pm — No Comments


Staff
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


Staff
PPMD’s #EverySingleOne Tour: Los Angeles, CA

Children’s Hospital Los Angeles (CHLA) marked our first stop of the New Year on PPMD’s 2017 Every Single One Tour, held on January 21, 2017. We were thrilled to not only be holding another tour stop at one of …

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Added by PPMD on February 15, 2017 at 10:30am — No Comments


Staff
Marathon Pharmaceuticals Pauses Commercialization of EMFLAZA™ (deflazacort)



UPDATE: February 13, 2017, 5:10 PM -- An additional statement from Marathon has been added to the blog



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Today, Marathon…

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Added by PPMD on February 13, 2017 at 4:00pm — No Comments


Staff
Deflazacort Approved in the U.S. for Duchenne

UPDATE 2/10: Marathon has released links to new resources, including the EMFLAZA website (https://emflaza.com) and a Patient Support FAQ document (…

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Added by Pat Furlong on February 9, 2017 at 5:00pm — 2 Comments


Staff
PPMD to Host Forum on Access to Emerging Therapies on February 13th, 2017

Following decades of strategic advocacy, research investment, and biopharmaceutical development, the U.S. Duchenne community recently arrived in the post-approval space and is poised to receive additional novel therapies from a robust innovation pipeline.…

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Added by PPMD on February 7, 2017 at 1:57pm — No Comments


Staff
PPMD’s Duchenne Drug Development Roundtable: Bringing Industry Together to Accelerate Research

Better. Faster. Now.

 

How do we apply the ‘regulatory learnings’ of the past 18 months back into our robust Duchenne pipeline to ensure as many effective therapies yield from our pipeline as possible?

Under the leadership of PPMD's Duchenne Drug Development Roundtable (DDDR), our community is determined to figure that out. Today is the first of a…

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Added by Abby Bronson on February 1, 2017 at 10:00am — No Comments


Staff
PPMD Awards $600,000 Grant to NJIT & Talem for Next Phase of Upper Extremity Exoskeleton

Robotics.

 

Ensuring that the worlds of teens and adults with Duchenne 'grow bigger', as they grow older by expanding their reach – both literally and figuratively.

  

Combing your hair. Lifting a glass to your mouth. Washing your hands. Pressing elevator…

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Added by PPMD on January 18, 2017 at 9:00am — 1 Comment


Staff
Meet the 2017 PPMD Adult Advisory Committee (PAAC)

Years ago, a friend of mine named Tom Bailey who had Duchenne wrote a memoir that he entitled, Moving Mountains Without Muscles. As I look at the tremendous accomplishments of PPMD’s Adult Advisory Committee since we first launched this leadership group 2 years ago, I am astounded by how…

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Added by Annie Kennedy on January 16, 2017 at 7:30pm — No Comments


Staff
ATTENTION: All Patients on Masters’ Deflazacort

As you know, the FDA is currently reviewing Marathon Pharmaceutical’s New Drug Application (NDA) for deflazacort for the treatment of Duchenne. It…

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Added by PPMD on January 16, 2017 at 6:30pm — No Comments


Staff
New Resource to Help Navigate EXONDYS 51 Access

Prior to the approval of EXONDYS 51™, we knew that access to emerging therapies would be a process – and an often challenging process at…

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Added by Annie Kennedy on January 13, 2017 at 11:00am — No Comments


Staff
New Research & Clinical Trial Tools on PPMD's DuchenneConnect!

We are excited to announce three new features now available on PPMD's DuchenneConnect! These tools are meant to help you in your journey as you gather information around potential research studies and potential new exon…

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Added by Ann Lucas on January 12, 2017 at 1:24pm — No Comments


Staff
Akashi Therapeutics Provides Update on Three Duchenne Compounds



This weekend, Akashi Therapeutics announced they are working toward having three novel, complementary compounds in the clinic in 2017 with potential to treat all Duchenne patients independent of their specific genetic…

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


Staff
Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program

On the heels of PPMD’s announcement last week of a $2.2 million grant to Nationwide Children’s Hospital’s gene therapy study being led by Dr. Jerry Mendell and Dr. Louise Rodino-Klapac, Sarepta Therapeutics today announced their commitment to the trial through a separate research agreement with Nationwide Children’s, including an…

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


Staff
PTC Provides Update on Next Steps for Translarna

In a corporate update released today, PTC Therapeutics announced next steps for their drug, Translarna in the U.S. According to the release, PTC plans to file the Translarna New Drug Application (NDA) for nmDMD over protest with the U.S. FDA in…

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Added by PPMD on January 9, 2017 at 10:30am — No Comments


Staff
PPMD Awards Nationwide Children’s Hospital $2.2 Million Grant to Explore Gene Therapy in Duchenne

Today, PPMD announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital.  Our purpose in awarding this grant is to open and accelerate the field of gene therapy – a strategy that seemed impossible as a potential…

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Added by Pat Furlong on January 6, 2017 at 9:00am — No Comments

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