Clinical trials: companies answer your questions

Clinical trials: companies answer your questions

Patients and families across the world know that clinical trials in neuromuscular diseases are now happening, and many of them wonder how they can get involved. Sometimes it can be a challenge to find out what all the different trials are, where they are happening, and who might be eligible. TREAT-NMD regularly receives questions from patients and families who want to find out whether they are eligible for the clinical trials that they have heard about. We therefore decided to put these questions to the pharmaceutical companies who are running these trials.

The first set of questions was sent to GSK, and we have posted the answers we received in this section. We hope these will help clarify some of the questions patients and families have about the DMD trials that GSK is running. In future we will also be asking similar questions of the other companies involved in trials for DMD and other neuromuscular diseases.

There is a lot to understand before deciding whether or not to take part in a clinical trial. You can read about some of the general principles behind clinical research here.

If you would like to comment on the GSK responses or suggest questions for other companies, please write to TREAT-NMD at

These questions have now been translated into Japanese and are available from the Japanese Registries website - Remudy.


12 Apr 2017