Evidence map›Paper›PMID 32076754›Full record

ReviewDer Nervenarzt2020

[Gene-specific treatment approaches in muscle diseases].

Diana Lehmann Urban, Ilka Schneider

Abstract readReview
PubMed Publisher
In one paragraph

Review in Der Nervenarzt, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.3field-weighted citation impact, top 44% 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

1 citing paper in PubMed, 5 citations in OpenAlex.

  1. Review
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.

Diana Lehmann UrbanNeurologische Universitätsklinik, Universitäts- und Rehabilitationskliniken Ulm, Oberer Eselsberg 45, 89081, Ulm, Deutschland. diana.lehmann@rku.de.
Ilka SchneiderNeurologie Klinikum St. Georg, Leipzig, Deutschland.
Klinikum St. Georg · DEUniversity and Rehabilitation Clinics Ulm · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Gene-specific treatment for hereditary muscle diseases has made great progress in recent years. The pathomechanisms of many of these diseases could be decrypted using molecular genetic techniques, paving the way for disease-modifying treatment options. A milestone was undoubtedly the successful translation of the antisense oligonucleotide (ASO) technology into clinical practice, with gene-specific ASOs being approved for the first time in 2016 for the treatment of spinal muscular atrophy and Duchenne muscular dystrophy. This article reviews recent developments in the field of antisense and gene therapies for hereditary muscle diseases.

Indexed as

Genetic TherapyMuscular DiseasesOligonucleotides, AntisenseHumansMuscular Atrophy, SpinalMuscular Dystrophy, DuchenneOligonucleotides, AntisenseAntisense oligonucleotideCell-based treatmentGene therapyMuscular dystrophy, DuchenneMyotonic dystrophy 1

Identifiers

PMID32076754
OpenAlexW3008074577

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.