Evidence map›Paper›PMID 36995385›Full record

ReviewDer Nervenarzt2023

[Update on drug treatment of spinal muscular atrophy].

Heike Kölbel, Tim Hagenacker

Abstract readEnglish AbstractReview
PubMed Publisher
In one paragraph

Review in Der Nervenarzt, 2023. 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, top 95% of its field
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, 0 citations in OpenAlex.

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

2 authors at 2 institutions in 1 country.

Heike KölbelKlinik für Kinderheilkunde, Center for Translational Neuro- and Behavioral Science, Universitätsmedizin Essen (UME), Universitätsmedizin Essen (UME), Essen, Hufelandstr. 55, 45147, Essen, Deutschland. heike.koelbel@uk-essen.de.
Tim HagenackerKlinik für Neurologie und Center for Translational Neuro- and Behavioral Science, Universitätsmedizin Essen (UME), Essen, Hufelandstr. 55, 45147, Essen, Deutschland. tim.hagenacker@uk-essen.de.
Essen University Hospital · DEZentrum für Kinderheilkunde · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThe 5q-associated spinal muscular atrophy (SMA) is a hereditary motor neuron disease leading to progressive tetraplegia, often involving the bulbopharyngeal and respiratory muscle groups. The disease usually manifests in early childhood and, if untreated, is progressive throughout life and associated with numerous complications depending on the severity. Since 2017, genetically based therapeutic mechanisms are now available that correct the causative deficiency of survival motor neuron (SMN) protein and lead to significant modifications in disease progression. As the number of treatment options increases, the question of which patient is suitable for which treatment also arises.

objectiveThis review article provides an update on the current treatment strategies for SMA in children and adults.

Indexed as

Muscular Atrophy, SpinalChildChild, PreschoolDisease ProgressionGenetic TherapyHumansGene therapyNewborn screeningNusinersenOnasemnogene abeparvovecRisdiplam

Identifiers

PMID36995385
OpenAlexW4361273894

What OpenQuestion holds

Textmetadata
Read underepoch 390

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.