Key result
As this is a protocol for a systematic review, no clinical findings or results are reported.
Why the study?
The objective was to analyse the benefits and harms of pharmacological or other interventions compared with placebo or standard care for RYR1-related disorders.
Do pharmacological or other interventions improve motor and respiratory function or reduce episode frequency in patients with RYR1-related disorders?
Systematic Review
Do pharmacological or other interventions improve motor and respiratory function or reduce episode frequency in patients with RYR1-related disorders?
This is a protocol for a Cochrane Review to evaluate the benefits and harms of interventions for RYR1-related disorders.
Evidence on interventions for RYR1-related disorders remains absent; protocol will synthesize future data on motor, respiratory, and rhabdomyolysis outcomes.
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.
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Raga et al. (2024) conducted a systematic review in RYR1-related disorders. Pharmacological or other interventions (e.g., special diet, exercise programme) vs. Placebo or standard care was evaluated on Improvement in motor and respiratory function and/or reduction in the frequency of episodes (exertional myalgia and rhabdomyolysis). As this is a protocol for a systematic review, no clinical findings or results are reported.
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