Clinical Trial Readiness


Effective Therapies

As part of its mission to bring effective therapies to patients in a timely & sustainable way, SMA Europe plays an important role in the development of treatments for SMA, through supporting, stimulating and promoting patient-relevant clinical trials.

We do this through our Treatment Committee and our Clinical Trials Readiness Project.

Our Treatment Committee

This committee is made-up of patient experts who work on projects related to treatment or represent SMA Europe in advisory boards with each industry involved in developing treatments for SMA.






Our Clinical Trials Readiness Project

Our Clinical Trials Readiness Project is part of a collaboration with Cure SMA and the Industry Consortium in the US, where we have been working on ensuring the readiness of centres in Europe and of people living with SMA to take up trials for SMA.

The Clinical Trial Readiness Project was initiated in 2018 and is designed to equip clinical trial sites with resources that support their ability to run or expand SMA clinical trials in a patient-centred way. This programme therefore includes the preparation and sharing of already available education materials to help sites and teams learn about SMA and offers tips for conducting clinical trials in SMA.

This project consists of different phases:

1: Understanding and expanding Europe’s current capacity to run clinical trials for potential SMA therapies. An analysis of the data obtained indicated that sites wished for educational material and training opportunities to increase their capacity to run clinical trials for SMA in their countries.


2: Training opportunities for clinicians and other health practitioners:

a) Sharing and adapting resources for European clinical trial centre use:

i) Our own infopacks:

ii) External educational resources:

b) Sponsored neurologists to attend a Treat-NMD masterclass on SMA

c) Organised a workshop on clinical trial best practices:

d) Sponsored Neurologists to attend our European Scientific and Clinical Congress


3: Educational activities for people either living with SMA or their carers, who wish to understand the processes involved in clinical trials and how best to participate. More information on this project will be available soon.