Long‐Term Effects of Nusinersen Dosing Frequency on Adult Patients With Spinal Muscular Atrophy: Efficacy of a 6‐Month Dosing Interval
The findings of this study suggest that the 6-month nusinersen dosing protocol provides sustained long-term benefits for ambulatory adult SMA patients. For nonambulatory patients, the 6-month protocol appears less effective than the 4-month protocol. We believe that future nusinersen treatment strategies for adult SMA patients should be flexible, with adjustments based on disease severity. In particular, increasing the dosing frequency and/or dosage in nonambulatory patients may lead to greater improvements.
Authors
- Hitaru Kishida (ORCID: https://orcid.org/0009-0004-5690-2721)
- Hiroshi Doi (ORCID: https://orcid.org/0000-0001-5807-2523)
- Hideyuki Takeuchi (ORCID: https://orcid.org/0000-0001-5912-7581)
- Yuichi Higashiyama
- Fumiaki Tanaka (ORCID: https://orcid.org/0000-0002-9961-2693)
- Misako Kunii (ORCID: https://orcid.org/0000-0002-3294-3009)
- Naohisa Ueda (ORCID: https://orcid.org/0000-0001-6610-058X)
- Keita Takahashi (ORCID: https://orcid.org/0000-0002-4950-1354)
- Yosuke Miyaji (ORCID: https://orcid.org/0000-0002-4482-965X)
Institutions
- Yokohama City University Medical Center (JP)
- Yokohama City University (JP)
Publication Details
- Journal
- Brain and Behavior
- Published
- 2025-05-01
- DOI
- https://doi.org/10.1002/brb3.70528
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Japan Society for the Promotion of Science