Give your opinion on a genetically modified medicine for the treatment of Duchenne muscular dystrophy.
The FPS Public Health and the FAMHP invite you to participate in the public consultation on a clinical trial of the genetically modified medicine RO7494222 (SRP-9001) for the treatment of Duchenne muscular dystrophy in patients under the age of four years. The public consultation runs from 26 October 2022 to 25 November 2022.
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 a genetically modified medicine.
The medicine SRP-9001 has been developed to treat the underlying biological cause of Duchenne muscular dystrophy by replacing the dysfunctional or missing dystrophin proteins in the cardiac and skeletal muscles, which are affected by this lethal degenerative disease, by a functionally shortened micro-dystrophin. This way, micro-dystrophin would address the root cause of the disease, alter the course of the disease and address an important unmet medical need.
The aim of the clinical trial is to evaluate the safety and the effect of SRP-9001 in patients, under the age of four years, with Duchenne muscular dystrophy.
The clinical trial will take place at the study hospital center of the Centre Hospitalier Régional de la Citadelle in Liège from the fourth quarter of 2022 (Q4 2022) and the end of the trial is expected in November 2032.
How to give your opinion?
The public consultation runs until 25 November 2022 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.