Give your opinion on a genetically modified medicine for the treatment of Duchenne muscular dystrophy

date: 17/01/2022

The FPS Public Health and the FAMHP invite you to participate in the public consultation on a clinical trial of the genetically modified medicine SRP-9001 for the treatment of Duchenne muscular dystrophy. The public consultation runs from 17 January 2022 to 16 February 2022.

For each clinical trial application for the use of a genetically modified organism, 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 effect of SRP-9001 on the functional motor skills of 120 young male patients aged between four and eight years, five of whom are in Belgium.

The clinical trial will take place at the University Hospital Ghent.

How to give your opinion?
The public consultation runs until 16 February 2022. You can access to various data from the application file, as well as an online form to comment or ask questions.

General information on GMO public consultation.

Last updated on 17/01/2022