Evidence map›Paper›PMID 42100011›Full record

ArticleThe Lancet regional health. Western Pacific2026

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

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In one paragraph

Article in The Lancet regional health. Western Pacific, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Emily E FarahDiscipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, University of New South Wales, Sydney, Australia.
Sarah-Grace PaguintoDiscipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, University of New South Wales, Sydney, Australia.
Melissa R MandarakasDiscipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, University of New South Wales, Sydney, Australia.
Karen HerbertDiscipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, University of New South Wales, Sydney, Australia.
Smriti KrishanDepartment of Psychology, Sydney Children's Hospital Randwick, Sydney, Australia.
Hugo SampaioDepartment of Neurology, Sydney Children's Hospital Randwick, Sydney, Australia.
Didu S KariyawasamDiscipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, University of New South Wales, Sydney, Australia.
Michelle A FarrarDiscipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, University of New South Wales, Sydney, Australia.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Despite diagnostic and therapeutic advances for spinal muscular atrophy (SMA), long-term outcomes focused on functional independence for children have not been explored. This study aimed to characterise adaptive functioning for affected children within the contemporary SMA treatment paradigm. Methods: This prospective, non-randomised Australian cohort study was conducted from January 1 to November 14 2025 and included children with SMA aged 4-12 years, diagnosed and treated through newborn screening (NBS) or clinical referral (CR). Adaptive functioning was primarily assessed using the Pediatric Evaluation of Disability Inventory Computer Adaptive Test. Findings: Thirty-nine children participated (NBS n = 18, CR n = 21) with median treatment duration of 67.3 months (IQR 54.0-85.0). Children had high levels of participation with scores within expected range for age-matched peers for social/cognitive (38/39, 97%), responsibility (35/39, 90%), daily activities (29/39, 74%) and mobility (16/39, 41%) domains. A greater proportion of children identified through NBS achieved expected scores for all domains compared to children diagnosed through CR (NBS 14/18 (78%), CR 2/21 (10%), p < 0.001). All children diagnosed through NBS with 3 survival motor neuron 2 gene ( Interpretation: Whilst heterogenous in terms of performance-based measures, children with SMA have collective strengths in social-cognitive and responsibility aspects of adaptive functioning. Early diagnosis and treatment through NBS and Funding: National Health and Medical Research Council Investigator Grant (1194940, 2026317), University of New South Wales (UGCA1064).

Indexed as

Adaptive functioningInternational classification of functioningPatient-reported outcomesSpinal muscular atrophy

Identifiers

PMID42100011
PMCPMC13146543

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.