Effectiveness and safety of Risdiplam for types 1–3 spinal muscular atrophy in a single center
Xiaomei Zhu, Hui Li, Chaoping Hu, Min Wu, Shuizhen Zhou, Yi Wang, Wenhui Li · 2025
Read Effectiveness and safety of Risdiplam for types 1–3 spinal muscular atrophy in a single center by Xiaomei Zhu, Hui Li, Chaoping Hu, Min Wu, Shuizhen Zhou, Yi Wang, Wenhui Li (2025) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-12849749.txt.
Description
Xiaomei Zhu and co-authors published this study in 2025. It reports real-world results for risdiplam, a daily oral drug for spinal muscular atrophy. Trials had shown the drug works; clinic data from China were scarce. One children's hospital followed twelve patients with types 1 to 3 of the disease, treated between 2021 and 2024. Motor scores, milestones, breathing, and feeding were tracked for eight to eighteen months.
Eleven of the twelve children improved in motor function. Nine reached new motor milestones, after about ten months on average. Younger children and those treated earlier gained the most. Side effects were mostly chest infections, blamed on the disease rather than the drug. No child stopped treatment or died. The authors back the drug's value in daily practice, and stress early diagnosis.
Catalog record
| CREATOR | Xiaomei Zhu, Hui Li, Chaoping Hu, Min Wu, Shuizhen Zhou, Yi Wang, Wenhui Li |
|---|---|
| YEAR | 2025 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 32 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |