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

date: 14/02/2024

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-9003 for the treatment of limb girdle muscular dystrophy. The public consultation runs from 14 February 2024 to 14 March 2024.

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.

Limb girdle muscular dystrophies (LGMDs) is a group of rare diseases that affect the muscles. LGMDs are caused by genetic mutations that cause errors in our body's instructions to make proteins that are important for muscle health. Without these proteins, people with LGMD have muscle loss and weakness that get worse over time.

In this clinical trial, the medicine SRP-9003 will be administered to patients from the     age of four years with disease type LGMD2E/R4. There is currently no treatment or cure for LGMD2E/R4. 
SRP-9003 has been developed to deliver a corrected version of the mutated gene that causes LGMD2E/R4. This would allow a patient with LGMD2E/R4 to make the relevant missing protein.

The aim of this trial is to assess the safety and efficacy of the medicine SRP-9003.

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

How to give your opinion?
The public consultation runs until 14 March 2024 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 14/02/2024