ArticleFrontiers in neurology2024
Motor and neurocognitive profiles of children with symptomatic spinal muscular atrophy type 1 with two copies of
Article in Frontiers in neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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.
Who cites it
11 citing papers in PubMed, 1 synthesis or guideline pooled it, 15 citations in OpenAlex.
- Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis.Gene therapy · 2025Pooled it
- Disease-Modifying Therapies in Spinal Muscular Atrophy: Neurodevelopmental and Behavioral Outcomes in the Treatment Era.Advances in therapy · 2026Review
- From Survival to Participation: Early Powered Mobility in the New Era of Spinal Muscular Atrophy Type I.Journal of clinical medicine · 2026Review
- Cognitive and neurodevelopmental outcomes in spinal muscular atrophy: a scoping review.Frontiers in cellular neuroscience · 2026Review
- Prenatal SMN-dependent defects in translation uncover reversible primary cilia phenotypes in spinal muscular atrophy.JCI insight · 2025Article
- Transforming Spinal Muscular Atrophy: From Pivotal Trials to Real-World Evidence and Future Therapeutic Frontiers in Types 1 and 2.Biomedicines · 2025Review
- Neurodevelopmental and mental disorders in children with type I and presymptomatic spinal muscular atrophy.Scientific reports · 2025Article
- Families' Perceptions of Powered Mobility for Participation in Children With Spinal Muscular Atrophy Type 1: A Photovoice Study.Health expectations : an international journal of public participation in health care and health policy · 2025Article
- Real-world outcomes of spinal muscular atrophy treatment with onasemnogene abeparvovec in Croatia: a comprehensive case series and literature review.Frontiers in medicine · 2025Article
- Onasemnogene-abeparvovec administration to premature infants with spinal muscular atrophy.Annals of clinical and translational neurology · 2024Article
- AMEsobreRuedas Early Powered Mobility in Children with Spinal Muscular Atrophy Type I: Protocol of a Randomized Controlled Trial.Journal of clinical medicine · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Introduction: Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by mutations in the survival motor neuron 1 ( Methods: This longitudinal observational study evaluated changes in motor and neurocognitive functioning over a 1-year period after administration of onasemnogene abeparvovec in 12 symptomatic SMA type 1 patients with two copies of Results and discussion: Statistically significant increases in median CHOP-INTEND scores from baseline were observed at 1, 3, 6, and 12 months after onasemnogene abeparvovec administration (all
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Registered trials
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.