Survival motor neuron protein is the optimal biomarker for evaluating the risdiplam treatment
Median SMN protein levels increased significantly after one month of risdiplam administration with some fluctuations. After five months of treatment, motor function, CMAP, and SMAIS-ULM caregiver scores improved. SMN protein is an appropriate biomarker for monitoring and evaluating the efficacy of risdiplam treatment in SMA.
Authors
- Hayato Suzuki (ORCID: https://orcid.org/0000-0001-6120-9471)
- Naoko Shima
- Kayoko Saito (ORCID: https://orcid.org/0000-0003-2632-0873)
- Moeka Shioya (ORCID: https://orcid.org/0000-0002-9787-3654)
- Tamaki Kato (ORCID: https://orcid.org/0000-0002-0148-2510)
- Kota Kajima
- Noriko Otsuki (ORCID: https://orcid.org/0000-0003-0848-6458)
- Mamoru Yokomura
- Hidetaka Wakabayshi
Institutions
- Toho University (JP)
- Tokyo Women's Medical University (JP)
Publication Details
- Journal
- Brain and Development
- Published
- 2025-08-11
- DOI
- https://doi.org/10.1016/j.braindev.2025.104410
- Citations
- 4
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 6.50
Funders
- Japan Agency for Medical Research and Development
- Ministry of Health, Labour and Welfare
- Japan Society for the Promotion of Science