February 15, 2018

FDA Publishes Finalized Duchenne Guidance for Drug Development

Today is a historic moment for the Duchenne community, as moments ago the FDA published the finalized “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” Guidance for Industry (click here to access), along with four other guidances….

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January 10, 2018

First Duchenne Patient Dosed in Microdystrophin Gene Therapy

“DMD gene therapy went well. It was started at 1:15 PM and ended at 2:27 PM”     On Thursday, January 4, I received this message from Dr. Jerry Mendell accompanied by the photo above – Dr….

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December 19, 2017

Understanding Currently Recruiting Gene Therapy Studies

For the last many months, we have all been watching gene therapy make headlines around the world, across various diseases. As you know, earlier this year PPMD launched a Gene Therapy Initiative to help support the exploration…

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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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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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