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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December 8, 2016

How PPMD’s Certified Duchenne Care Centers Are Improving Quality & Access to Care

When I joined PPMD in 2012, care for Duchenne was all over the map. Sadly, there were over 150 clinics spread across the US, but there was no oversight of these clinics – the community…

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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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July 20, 2015

PPMD Awards $70,000 Grant to Talem Technologies as Part of Robotics Initiative

Grant Will Be Used to Support Pilot Study for X-Ar Exoskeletal Arm Technology Parent Project Muscular Dystrophy has awarded Talem Technologies a grant for over $70,000 to support a pilot study of their X-Ar exoskeletal arm technology. The grant…

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April 28, 2015

Let’s Get Them Moving Workshop: Our First Steps Toward That Goal

Yesterday, Parent Project Muscular Dystrophy and Duchenne Parent Project-Netherlands (DPP) co-hosted the first ever conference on assistive technology for Duchenne muscular dystrophy. There were twenty-five participants in the workshop, from six countries, representing academia (both researchers and…

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