May 3, 2021

Thank you for helping turn obstacles into possibilities!

Thank you to every person who donated over the past month to support PPMD’s Gene Therapy Initiative. Thanks to your generosity, we can continue answering the difficult questions we face when it comes to the…

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May 3, 2021

Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051

Sarepta has announced positive results from Part A of the MOMENTUM study (Study 5051-201), a Phase 2 clinical trial of SRP-5051, its next-generation treatment for individuals with Duchenne who are amenable to exon 51 skipping. Part…

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April 29, 2021

Safety Preparedness Toolkit for People with Duchenne

If we have learned anything over the past year, it is that life is unpredictable. Most of us did not anticipate a global pandemic or record-breaking freezing temperatures in Texas leading to a power crisis….

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April 28, 2021

Santhera and ReveraGen Announce New 2.5-year Treatment Data with Vamorolone in Duchenne

Today Santhera and ReveraGen shared 2.5-year treatment data with vamorolone in Duchenne. In the announcement below the companies reported on data from 46 patients who have participated in two open-label extension studies covering 2.5 years,…

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April 27, 2021

C-Path Opens Access to Duchenne Regulatory Science Consortium (D-RSC) Database

PPMD is proud to be an original funder and continued supporter of the Duchenne Regulatory Science Consortium (D-RSC) database, which includes data from Duchenne clinical trials, natural history studies, and clinical data collections. With the strength…

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April 27, 2021

Only a few days left to have your gift to PPMD matched!

When I think about Gene Therapy, I think about the possibilities. I think about those who fight every day so that Gene Therapy may become a safe reality for many. I think about a future…

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April 26, 2021

Webinar Q&A: Gene Therapy – Turning Obstacles into Possibilities

Last week, PPMD held a webinar to discuss the lessons learned in Gene Therapy, current challenges for our community, and what the future holds for these promising treatments. After opening with an insightful presentation from…

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April 23, 2021

Exciting News! Your gift will be Matched Dollar-for-Dollar up to $50,000

There is nothing ordinary about Duchenne. We all know that. Our Duchenne story started in the ER. At two years old, my middle son was involved in a serious accident and went into cardiac arrest….

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April 21, 2021

WATCH: Gene Therapy – Turning Obstacles into Possibilities (Webinar Recording)

On April 20, 2021, PPMD’s Pat Furlong & Eric Camino were joined by Drs. Nagaraju and Shieh for an interactive panel discussion on the lessons learned in Gene Therapy, current challenges for our community, and…

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April 15, 2021

PPMD’s End Duchenne Tour turns 5!

In late 2015, we had a discussion at the PPMD office in Hackensack. Was it time to kick our local educational events up a notch? Have we certified enough centers to have a “home base”…

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