December 23, 2019

Sarepta Announces Partnership with Roche Outside the U.S. for its Investigational Micro-dystrophin Gene Therapy

Sarepta Therapeutics, Inc. today announced that Sarepta and Roche have entered into a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, outside the United States….

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December 20, 2019

Sarepta Therapeutics Shares Winter 2019 Newsletter

Sarepta has shared the Winter 2019 issue of their newsletter, including updates on current Sarepta programs. Click here to download.

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December 18, 2019

PPMD Awards $465,000 Grant to The Ohio State University to Rescue Cardiac Dystrophin & Function in Duchenne

Parent Project Muscular Dystrophy is excited to announce that Renzhi Han, an associate professor at The Ohio State University College of Medicine, will receive a $465,000 grant to study the potential use of in vivo…

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December 18, 2019

Solid Biosciences Provides Data Update from SGT-001 Development Program

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that the status of the sixth patient dosed in the higher 2E/14 vg/kg, who experienced the…

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December 4, 2019

Astellas Enters into Definitive Agreement to Acquire Audentes Therapeutics

Astellas Pharma and Audentes Therapeutics announced that they have entered into a definitive agreement for Astellas to acquire Audentes. We look forward to learning more as this partnership moves forward. Read the Announcement from Audentes…

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November 12, 2019

Solid Biosciences Provides SGT-001 Program Update

Solid Biosciences Inc. provided a clinical update on SGT-001 and reported that the FDA has notified the company that IGNITE DMD, its Phase I/II study of SGT-001, has been placed on clinical hold. To date,…

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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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