Risdiplam in Adult Patients With 5q Spinal Muscular Atrophy: A Single‐Center Longitudinal Study
Risdiplam treatment over 30 months resulted in overall CMI in most treated adult SMA type 2 or 3 patients. Outpatient drug administration and overall patient management proved feasible and safe. Larger studies are warranted.
Authors
- Maria Gavriilaki (ORCID: https://orcid.org/0000-0002-1958-0063)
- Marianthi Arnaoutoglou (ORCID: https://orcid.org/0000-0001-6476-2651)
- Maria Moschou (ORCID: https://orcid.org/0000-0002-1175-7506)
- Vasilios Κ. Kimiskidis (ORCID: https://orcid.org/0000-0002-3335-3019)
- Maria Pagiantza
Institutions
- Aristotle University of Thessaloniki (GR)
- AHEPA University Hospital (GR)
Publication Details
- Journal
- Muscle & Nerve
- Published
- 2024-12-26
- DOI
- https://doi.org/10.1002/mus.28327
- Citations
- 9
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 3.94