A Randomized, Double-Blind, Placebo-Controlled, Gene-Delivery Clinical Trial of rAAVrh74.MHCK7.micro-dystrophin for Duchenne Muscular Dystrophy

rAAVrh74.MHCK7.micro-dystrophin (SRP-9001) was designed to safely express a functional micro-dystrophin protein in skeletal and cardiac muscle. The purpose of Study 102 is to evaluate the safety and efficacy of IV SRP-9001 in people with DMD by measuring biological and clinical endpoints in a Phase 2 multicenter, randomized, double-blind, placebo-controlled trial (NCT03769116). Results from the 41 patients that have been randomized and dosed (SRP-9001 or placebo) in Part 1 will be presented.

{{assetTitle}}

Authors {{assetAuthors}}

Published date{{formattedPublishDate}}

{{assetDescription}}

Welcome to Medically

The Roche Science Hub

This website is a non-promotional global resource intended to facilitate transparent scientific exchange regarding developments in medical research, diagnostics, and disease management. It is intended for healthcare professionals.

Not a healthcare professional? Browse:

This global website is intended for healthcare professionals outside the UK, US, Canada and Australia. The content on this website may include scientific information about experimental or investigational compounds, indications and services that are not approved or valid in your jurisdiction. Registration status and prescribing information of medicinal products may differ between countries. Please refer to local product information for any medicinal products mentioned on this website. Information available on this website does not constitute professional medical advice, and Roche and Genentech accept no responsibility for access to or use of the same.

You are Leaving Medically

By following this link, you are leaving Roche Website and entering a site that is not owned or controlled by Roche. Roche does not take any responsibility for acces to or use of this website, nor for any content therein.