January 2017 Blog Posts (13)

PPMD Designates Yale New Haven Children's Hospital a Certified Duchenne Care Center

Yale New Haven Children's Hospital's Muscular Dystrophy Program became the 13th clinic named a Certified Duchenne…


Added by PPMD on January 19, 2017 at 11:30am — No Comments

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



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 buttons. Zipping a coat. Opening a door. Turning on a light.…


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

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 much passion, expertise, and effort each member has dedicated…


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

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 is our understanding that a decision is expected to be made…


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

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…


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

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 skipping therapies.  …


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

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 mutation.

This is promising news for a…


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

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…


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

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…


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

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 treatment for Duchenne as far back as the 1990s. If successful,…


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

Two simple words...THANK YOU!

We not only met our goal, we surpassed it!

We could not be more overwhelmed by the incredible response to our holiday campaign. In many ways, with a therapeutic pipeline full of promising treatments, it feels like the future of Duchenne therapy is here. Thanks to this community, we head into 2017 with…


Added by Pat Furlong on January 4, 2017 at 3:47pm — No Comments

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