Evidence mapPaperPMID 39201450Full record

ArticleInternational journal of molecular sciences2024

Impact of Disease Severity and Disease-Modifying Therapies on Myostatin Levels in SMA Patients.

Laurane Mackels, Virginie Mariot, Laura Buscemi, Laurent Servais, Julie Dumonceaux

Abstract read
In one paragraph

Article in International journal of molecular sciences, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 pooled it
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

5 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Article
  4. Article
  5. 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

5 authors.

Laurane MackelsAdult Neurology Department, Citadelle Hospital, 1 Boulevard Du 12e De Ligne, 4000 Liege, Belgium.ORCID 0000-0002-2132-3736
Virginie MariotNIHR Great Ormond Street Hospital Biomedical Research Centre and Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.ORCID 0000-0002-3222-9286
Laura BuscemiNeuromuscular Center, Citadelle Hospital, 1 Boulevard Du 12e De Ligne, 4000 Liege, Belgium.
Laurent ServaisMDUK Oxford Neuromuscular Center, Department of Paediatrics, NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford OX3 9DU, UK.ORCID 0000-0001-9270-4061
Julie DumonceauxNIHR Great Ormond Street Hospital Biomedical Research Centre and Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.ORCID 0000-0002-2052-9361

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Clinical trials with treatments inhibiting myostatin pathways to increase muscle mass are currently ongoing in spinal muscular atrophy. Given evidence of potential myostatin pathway downregulation in Spinal Muscular Atrophy (SMA), restoring sufficient myostatin levels using disease-modifying treatments (DMTs) might arguably be necessary prior to considering myostatin inhibitors as an add-on treatment. This retrospective study assessed pre-treatment myostatin and follistatin levels' correlation with disease severity and explored their alteration by disease-modifying treatment in SMA. We retrospectively collected clinical characteristics, motor scores, and mysotatin and follistatin levels between 2018 and 2020 in 25 Belgian patients with SMA (SMA1 (

Indexed as

FollistatinMuscular Atrophy, SpinalMyostatinSeverity of Illness IndexAdolescentChildChild, PreschoolFemaleHumansInfantMaleOligonucleotidesRetrospective StudiesFollistatinMSTN protein, humanMyostatinnusinersenOligonucleotidesclinical trialsdisease-modifying therapiesfollisatinFSTNGDF8myostatinnusinersenspinal muscular atrophy

Identifiers

PMID39201450
PMCPMC11354404

What Socratic holds

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LicenceCC BY
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 Socratic graph.