Evidence map›Paper›PMID 39655671›Full record

ArticleThe Cochrane database of systematic reviews2024

Treatments for RYR1-related disorders.

Sharika Raga, Nicol Voermans, Ivan Perez-Neri, Jim Dowling, Heinz Jungbluth, Giovanni Baranello, Laurent Servais, Alice Tillema, Jo Wilmshurst

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in The Cochrane database of systematic reviews, 2024. 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
–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

1 citing paper in PubMed.

  1. Exercise capacity in RYR1-related myopathies.Orphanet journal of rare diseases · 2025
    Trial
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

9 authors.

Sharika RagaDepartment of Paediatric Neurology, University of Cape Town, Cape Town, South Africa.
Nicol VoermansNeurology, Radboud University Medical Center, Nijmegen, Netherlands.
Ivan Perez-NeriEvidence Synthesis Unit, National Institute of Rehabilitation Luis Guillermo Ibarra Ibarra, Mexico City, Mexico.
Jim DowlingDivision of Neurology, The Hospital for Sick Children, University of Toronto, Toronto, Canada.
Heinz JungbluthDepartment of Paediatric Neurology, Neuromuscular Service, Evelina's Children Hospital, Guy's & St. Thomas' Hospital NHS Foundation Trust, London, UK.
Giovanni BaranelloDevelopmental Neurosciences Department, UCL GOS Institute of Child Health, London, UK.
Laurent ServaisDepartment of Paediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK.
Alice TillemaMedical Library, Radboud University, Nijmegen, Netherlands.
Jo WilmshurstDepartment of Paediatric Neurology, University of Cape Town, Cape Town, South Africa.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesThis is a protocol for a Cochrane Review (intervention). The objectives are as follows: Primary objective To analyse the benefits and harms of pharmacological or other interventions (e.g. special diet, exercise programme) compared with placebo or standard care for RYR1-related disorders, including both permanent myopathies and intermittent (episodic) presentations (exertional myalgia and rhabdomyolysis), with the aim to improve motor and respiratory function and/or to reduce the frequency of episodes, respectively. Secondary objectives To assess whether the interventions, compared with placebo or standard of care, change the outcome of RYR1-related diseases. To assess whether the interventions, compared with placebo or usual care, change the expression of the disease state in patients with RYR1-related diseases. To identify a set of standardised outcome tools to be used in future studies.

Indexed as

Muscular DiseasesRhabdomyolysisRyanodine Receptor Calcium Release ChannelHumansMyalgiaRandomized Controlled Trials as TopicSystematic Reviews as TopicRyanodine Receptor Calcium Release ChannelRYR1 protein, human

Identifiers

PMID39655671
PMCPMC11629463

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.