December 13, 2017

Understanding Gene Therapy: The Screening Process

With advancements made in Duchenne gene therapies now coming to fruition, a number of questions are being asked about who may qualify for the upcoming trials. While the inclusion criteria of each trial will be different based…

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November 15, 2017

Race Athlete Spotlight: Katie Napoli

Thirteen year old Katie Napoli is one of our youngest “veteran” runners, having been with the team since she was 8 years old. Katie and her family run for Katie’s cousin, Danny, and are part…

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September 30, 2017

PPMD Dream Auction to End Duchenne

On September 23rd, the Hester and Zenobio families gathered with their friends and family for the PPMD DREAM Auction in Tacoma WA. With over 400 guests, they raised close to $400,000 for Duchenne research.  …

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April 5, 2017

PPMD’s Duchenne Drug Development Roundtable Meeting Series, Part 3: Clinical Trial Readiness from the Viewpoint of Clinicians and Infrastructure

Accelerating trials. Protecting our robust therapeutic pipeline. Ensure as many hits on goal as we can. Priorities shared by all of us. And to achieve this, PPMD has convened our industry partners through our Duchenne Drug…

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March 3, 2017

PPMD’s Duchenne Drug Development Roundtable Meeting Series, Part 2: Optimizing Clinical Trials

This week, PPMD convened Duchenne industry partners, experts in adaptive trial design and biostatistics, and innovative partners from outside of the Duchenne world who’ve revolutionized their clinical trial spaces for the second meeting of our Duchenne…

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February 1, 2017

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…

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January 30, 2017

PPMD Awards $250,000 Grant to UT Southwestern Medical Center to Explore CRISPR/Cas9 Technology

PPMD today announced a $250,000 grant to be awarded to Dr. Eric Olson and the Department of Molecular Biology at UT Southwestern Medical Center. This grant, part of PPMD’s Gene Transfer Initiative, will support Dr. Olson’s ongoing study of CRISPR/Cas9…

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January 18, 2017

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….

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January 6, 2017

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…

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November 14, 2016

Duchenne Carrier Study at Nationwide Children’s Hospital

Frequently, I get calls from mothers/sisters/aunts of people living with Duchenne about issues related to being a carrier of Duchenne: Should they have carrier testing? Why should they have carrier testing? If they are carriers, what…

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