Adaptive functioning in school-aged children with spinal muscular atrophy in the treatment era: a non-randomised cohort study
Emily E. Farah, Sarah-Grace Paguinto, Melissa R. Mandarakas, Karen Herbert, Smriti Krishan, Hugo Sampaio, Didu S. Kariyawasam, Michelle A. Farrar · 2026
Read Adaptive functioning in school-aged children with spinal muscular atrophy in the treatment era: a non-randomised cohort study by Emily E. Farah, Sarah-Grace Paguinto, Melissa R. Mandarakas, Karen Herbert, Smriti Krishan, Hugo Sampaio, Didu S. Kariyawasam, Michelle A. Farrar (2026) 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-13146543.txt.
Description
Emily E Farah and colleagues report a two thousand twenty-six Australian cohort study of daily functioning in school children with spinal muscular atrophy. New treatments have transformed this once-deadly muscle disease. How independent treated children are in everyday life was unexplored.
Children aged four to twelve were found through newborn screening or later clinical referral. A standard disability inventory measured social, responsibility, daily activity, and mobility skills. Most children scored as expected for their age in social and responsibility areas. Mobility lagged, with fewer than half at expected levels. Screening-found children did better across all areas.
Catalog record
| CREATOR | Emily E. Farah, Sarah-Grace Paguinto, Melissa R. Mandarakas, Karen Herbert, Smriti Krishan, Hugo Sampaio, Didu S. Kariyawasam, Michelle A. Farrar |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 64 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |