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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.

Original publication

DOI

10.1002/14651858.CD014439

Type

Journal

Cochrane Database Syst Rev

Publication Date

10/12/2024

Volume

12

Keywords

Humans, Muscular Diseases, Myalgia, Randomized Controlled Trials as Topic, Rhabdomyolysis, Ryanodine Receptor Calcium Release Channel, Systematic Reviews as Topic