Information material

Our Clinical Trial Readiness Project 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.

As part of the provision of educational material, we highlight the following resources:

Our own information packs:

  1. SMA: pathology, diagnosis, clinical presentation, therapeutic strategies and treatments;
  2. Standards of care – Available soon
  3. Conducting clinical trials – Available soon

External educational resources:

  1. European Reference Networks: Educational resources
  2. Treat-NMD: Facing the Challenges of Clinical Trials
  3. EUPATI: Patient engagement and Toolbox