May 17, 2022

Nationwide Children’s Announces Restoration of Full-Length Dystrophin Using Dup 2 Gene Therapy Approach

PPMD is very excited to see promising results shared from Nationwide Children’s scAAV9.U7.ACCA gene therapy trial, a Phase 1/2 study aiming to induce exon skipping for individuals with a duplication of exon 2 to restore full-length…

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April 17, 2019

PPMD Awards $100,000 Grant to Nationwide Children’s Hospital to Further Explore GALGT2 Gene Therapy

PPMD is excited to announce that Drs. Paul Martin and Kevin Flanigan from the Research Institute at Nationwide Children’s Hospital have completed work under a $100,000 grant exploring GALGT2 gene therapy technology as a potential treatment…

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October 4, 2018

Jerry Mendell, M.D. Shares Positive Updated Results from Gene Therapy Micro-dystrophin Trial

  PPMD is excited to learn that Dr. Jerry Mendell of Nationwide Children’s Hospital today shared positive results for the fourth patient in Sarepta’s micro-dystrophin trial, indicating positive functional signals for all of the patients….

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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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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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