Evidence map›Paper›PMID 42352652›Full record

ArticleBrain sciences2026

Longitudinal Transcriptomic Analysis Reveals Systemic Effects of Risdiplam in Adults with Spinal Muscular Atrophy.

Maria Liguori, Arianna Consiglio, Eustachio D'Errico, Ylenia Antonacci, Martina Coffa, Alessandro Introna, Isabella Laura Simone

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In one paragraph

Article in Brain sciences, 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
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0citing papers in PubMed
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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

7 authors.

Maria LiguoriNational Research Council, Department of Biomedicine, Institute of Biomedical Technologies, Bari Unit, 70125 Bari, Italy.ORCID 0000-0003-4338-0392
Arianna ConsiglioNational Research Council, Department of Biomedicine, Institute of Biomedical Technologies, Bari Unit, 70125 Bari, Italy.
Eustachio D'ErricoNeurology Unit, Department of Translational Biomedicine and Neuroscience, University of Bari "Aldo Moro", 70124 Bari, Italy.ORCID 0000-0002-3762-5800
Ylenia AntonacciNational Research Council, Department of Biomedicine, Institute of Biomedical Technologies, Bari Unit, 70125 Bari, Italy.ORCID 0009-0007-9546-6231
Martina CoffaNational Research Council, Department of Biomedicine, Institute of Biomedical Technologies, Bari Unit, 70125 Bari, Italy.
Alessandro IntronaNeurology Unit, Department of Translational Biomedicine and Neuroscience, University of Bari "Aldo Moro", 70124 Bari, Italy.ORCID 0000-0002-3141-6442
Isabella Laura SimoneSchool of Medicine, University of Bari "Aldo Moro", 70124 Bari, Italy.ORCID 0000-0002-7429-3091

Funding

Regione Puglia (D.U.P. n.246/2019, D.D. n. 3 of 13 January 2021)
6 · The paper itself

Abstract

backgroundSpinal Muscular Atrophy (SMA) is a neurodegenerative disease caused by reduced survival motor neuron (SMN) protein levels due to

methodsWe performed longitudinal RNA sequencing on peripheral blood samples from 16 adult SMA patients (types II and III) before and after 12 months of Risdiplam treatment to assess transcriptomic changes.

resultsDuring Risdiplam treatment, increased

conclusionsThese findings support Risdiplam's systemic impact in adult SMA subjects and reveal molecular distinctions between SMA phenotypes (types II and III), which may be of some relevance for future clinical and therapeutic strategies.

Indexed as

RisdiplamRNA sequencingSMN2 genespinal muscular atrophyTranscriptomics

Identifiers

PMID42352652
PMCPMC13297622

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