Longitudinal Efficacy of Nusinersen Treatment on Health‐Related Quality of Life and Independence in Children With Later‐Onset Spinal Muscular Atrophy
This study demonstrates the longitudinal effects of nusinersen treatment on multifaceted aspects of SMA patients, as captured by patient-reported outcome measures (PROMs). The inclusion of PROMs should be considered as part of the SMA multidisciplinary assessment.
Authors
- Shanshan Mao (ORCID: https://orcid.org/0000-0001-6736-1407)
- Dongming Zhou (ORCID: https://orcid.org/0000-0002-5989-7591)
- Feng Gao (ORCID: https://orcid.org/0000-0002-4902-7429)
- Yue Yan (ORCID: https://orcid.org/0000-0003-1387-4973)
- Yicheng Yu
- Liya Jiang
- Mei Yao (ORCID: https://orcid.org/0009-0001-2034-5108)
- Siyi Huang
- Yijie Feng
Institutions
- Children's Hospital of Zhejiang University (CN)
Publication Details
- Journal
- Muscle & Nerve
- Published
- 2024-12-24
- DOI
- https://doi.org/10.1002/mus.28311
- Citations
- 5
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 2.19
Funders
- National Natural Science Foundation of China
- Fundamental Research Funds for the Central Universities
- Zhejiang Province Public Welfare Technology Application Research Project