共 50 条
- [2] An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, 2022, 9 (06): : 810 - 818
- [4] Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study Orphanet Journal of Rare Diseases, 15