Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study.

From the caregivers' perspective, there was no alternative to participating in the EAP for nusinersen. All participants were positive regarding their decision to participate. However, this study suggests that developing procedures to increase speed and transparency and to ensure fairness could help to further improve the system of EAPs as a way to provide urgently needed care to highly vulnerable patients.