Spinal Muscular Atrophy: The Reality of the Adult Patient in Spain
These results provide evidence of a progressive impairment and increased sensitivity to treatment discontinuations in the SMA adult population, along with delayed or reduced access to commercialised SMA drugs and clinical trials. A more rigorous evaluation of the disease's impact and treatment benefits in the adult SMA population is necessary.
Authors
- Mencía de Lemus
- Maria Grazia Cattinari (ORCID: https://orcid.org/0000-0001-9821-4194)
- Eduardo F. Tizzano (ORCID: https://orcid.org/0000-0002-7116-6310)
- M Dumont
Institutions
- European Medicines Agency (NL)
- Vall d'Hebron Institut de Recerca (ES)
- Centre for Biomedical Network Research on Rare Diseases (ES)
Publication Details
- Journal
- Revista de Neurología
- Published
- 2025-03-27
- DOI
- https://doi.org/10.31083/rn33462
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00