gene therapy

October 7, 2019

2019 World Muscle Society Update – Days 2 & 3

This week, PPMD’s Abby Bronson, SVP of Research Strategy and Eric Camino, PhD, Director of Research and Clinical Innovation are attending the annual World Muscle Society (WMS) meeting, this year in Copenhagen, Denmark. Below, Abby…

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October 2, 2019

Duchenne UK and PPMD Award $200,000 Grant to Develop an Accepted Set of Patient Reported Outcomes

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK announced the joint funding of a project to create a set of agreed and validated Patient Reported Outcomes (PROs) for use in Europe and the US. The…

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September 25, 2019

WATCH: Introduction to Gene Therapy [PPMD Gene Therapy Webinar Series]

For PPMD a pivotal aspect of care is education. Without the proper knowledge of what the potential benefits and risks are of an investigational product a family cannot make well informed decisions. As the landscape…

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September 17, 2019

PPMD Awards University of Florida $1 Million for Novel Gene Therapy Approach Targeting the Heart

As you know, one of PPMD’s priorities in the fight to end Duchenne is maintaining cardiac health and preventing heart disease or dilated cardiomyopathy (DCM), better known as heart failure in Duchenne muscular dystrophy. Heart…

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September 5, 2019

Sarepta Therapeutics Shares Summer 2019 Newsletter

Sarepta has shared the Summer 2019 issue of their newsletter, including clinical updates. Click here to download.

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August 14, 2019

Solid Biosciences Provides Update on IGNITE DMD Clinical Trial

Today, Solid announced that the second patient has been dosed in the higher dose cohort of the IGNITE DMD Phase I/II clinical trial of SGT-001, the company’s AAV gene transfer candidate under investigation for Duchenne….

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Sarepta Therapeutics Comments on Erroneous Submission to US FDA Adverse Event Reporting System (FAERS)

Sarepta Therapeutics was informed earlier today that an adverse event report was submitted to the FDA’s adverse event reporting system (FAERs), a post-marketing surveillance database for approved therapies. Sarepta states that based on their investigation…

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Audentes Therapeutics Announces Plans to Commence Patient Dosing with AT702 in Q4 2019

Audentes Therapeutics has announced plans to commence patient dosing in the fourth quarter of 2019 with AT702 produced by Nationwide Children’s Hospital. AT702 is designed to induce exon 2 skipping to treat Duchenne caused by…

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PPMD Awards $175,000 Grant to UCLA to Explore Optimization of CRISPR Technology in Duchenne

Parent Project Muscular Dystrophy (PPMD) is excited to announce that Drs. Melissa Spencer and April Pyle from the David Geffen School of Medicine at UCLA and the Center for Duchenne Muscular Dystrophy at University of…

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Solid Biosciences Provides Update on IGNITE DMD Status and New Clinical Sites

Solid Biosciences shared an update on the progress of their IGNITE DMD Phase I/II clinical trial for investigational microdystrophin gene transfer, SGT-001. The update includes news about initiating dosing for their higher dose cohort of…

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