Give your opinion on a genetically modified medicine for the treatment of Duchenne Muscular Dystrophy

date: 29/03/2023

The FPS Public Health and the FAMHP invite you to participate in the public consultation on a clinical trial testing the genetically modified medicine RO7494222 (SRP-9001) for the treatment of Duchenne muscular dystrophy. The public consultation runs from 29 March to 28 April 2023.

For each clinical trial application for the use of a genetically modified organisms, the government organises a thirty-day public consultation, in accordance with the Royal Decree of 21 February 2005. As a citizen, you can give your opinion on this clinical trial application with the genetically modified medicinal product SRP-9001.

The primary objective of this study is to evaluate the effect of SRP-9001 on the physical function by assessing the performance of the upper limb (PUL).

Duchenne muscular dystrophy is caused by a mutation in the dystrophin gene, a key muscle protein. SRP-9001 is a gene therapy designed to replace the dysfunctional or missing dystrophin protein with a functional truncated dystrophin, called micro-dystrophin. The key affected tissues in this lethal degenerative disease are the heart and skeletal muscles. Thus, micro-dystrophin would address the root cause of Duchenne muscular dystrophy, alter the course of the disease, and address a significant unmet medical need.

The clinical trial will take place at the University Hospital UZ-Gent in Gent between 2023 and 2026.

How to give your opinion?
The public consultation runs until 28 April 2023 inclusive. You can access the various data in the application file, as well as an online form to send your comments or ask your questions.

General information on GMO - public consultation.
 

Last updated on 29/03/2023