Raga, S;
Voermans, N;
Perez-Neri, I;
Dowling, J;
Jungbluth, H;
Baranello, G;
Servais, L;
... Wilmshurst, J; + view all
(2024)
Treatments for RYR1-related disorders.
Cochrane Database of Systematic Reviews
, 2024
(12)
, Article CD014439. 10.1002/14651858.CD014439.
![[thumbnail of Raga_et_al-2024-Cochrane_Database_of_Systematic_Reviews.pdf]](https://discovery-pp.ucl.ac.uk/style/images/fileicons/text.png) |
Text
Raga_et_al-2024-Cochrane_Database_of_Systematic_Reviews.pdf - Published Version Access restricted to UCL open access staff until 11 December 2025. Download (214kB) |
Abstract
Objectives: This 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.
| Type: | Article |
|---|---|
| Title: | Treatments for RYR1-related disorders |
| Location: | England |
| DOI: | 10.1002/14651858.CD014439 |
| Publisher version: | https://doi.org/10.1002/14651858.cd014439 |
| Language: | English |
| Additional information: | This version is the version of record. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Humans, Muscular Diseases, Myalgia, Randomized Controlled Trials as Topic, Rhabdomyolysis, Ryanodine Receptor Calcium Release Channel, Systematic Reviews as Topic |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/10202856 |
Archive Staff Only
 |
View Item |
