Evidence map›Paper›PMID 41718941›Full record

ArticleAdvances in therapy2026

Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series.

Melissa D Svoboda, Nancy Kuntz, Carmen Leon-Astudillo, Barry J Byrne, Jena Krueger, Jennifer M Kwon, Cory Sieburg, Diana Castro

Abstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Advances in therapy, 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.

Melissa D SvobodaDivision of Pediatric Neurology/Neurodevelopment, Department of Pediatrics, CHRISTUS Children's/Baylor College of Medicine, San Antonio, TX, USA. melissa.svoboda@bcm.edu.
Nancy KuntzDivision of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
Carmen Leon-AstudilloDepartment of Pediatrics, Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.
Barry J ByrneDepartment of Pediatrics, Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.
Jena KruegerDivision of Pediatric Neurology, Department of Pediatrics, Helen DeVos Children's Hospital, Grand Rapids, MI, USA.
Jennifer M KwonDivision of Pediatric Neurology, Department of Neurology, University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI, USA.
Cory SieburgDivision of Pediatric Neurology, Department of Neurology, University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI, USA.
Diana CastroNeurology Rare Disease Center, Denton, TX, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

This Summary of Research summarizes a previously published original article, "Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series." Spinal muscular atrophy (SMA) is a rare genetic disease that causes muscle weakness and is associated with swallowing and breathing difficulties. Risdiplam (EVRYSDI

Indexed as

Muscular Atrophy, SpinalPyrimidinesSpinal Muscular Atrophies of ChildhoodAzo CompoundsChildChild, PreschoolFemaleGene Therapy AgentsHumansInfantMaleRecombinant Fusion ProteinsTreatment OutcomeAzo Compoundsonasemnogene abeparvovecPyrimidinesRecombinant Fusion ProteinsRisdiplamCase seriesGene therapyOnasemnogene abeparvovecRisdiplamSpinal muscular atrophy

Identifiers

PMID41718941
PMCPMC13065540

What OpenQuestion holds

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Registered trials

None linked

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.