As part of our mission to bring effective therapies to patients in a timely & sustainable way, we joined a Clinical Trial Readiness initiative developed by Cure SMA and its Industry Consortium in the U.S.
This project aims to understand and expand Europe’s current capacity to run clinical trials for potential SMA therapies.
In order to achieve this, several initiatives have been undertaken, including a survey to better understand the current landscape in Europe. An analysis of the data obtained indicated that sites would value educational material and training opportunities to increase their capacity to run clinical trials for SMA in their countries. We have therefore engaged in the following activities:
- Sharing and adapting resources for European clinical trial centre use
- Sponsoring Neurologists to attend Treat-NMD’s SMA Masterclass
- Organising a workshop on SMA clinical trial best practice
- Sponsoring Neurologists to attend our European Scientific and Clinical Congress 2020
We have now produced 1 of 3 information booklets, which are designed to equip clinical trial sites with resources that aim to support their ability to run or expand SMA clinical trials in a patient-centred way. This first booklet contains comprehensive information on SMA: the pathology, diagnosis, clinical presentation, therapeutic strategies and treatments which we are excited to share with you.