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- Sharika Raga, Nicol Voermans, Ivan Perez-Neri, Jim Dowling, Heinz Jungbluth, Giovanni Baranello, Laurent Servais, Alice Tillema, and Jo Wilmshurst.
- Department of Paediatric Neurology, University of Cape Town, Cape Town, South Africa.
- Cochrane Db Syst Rev. 2024 Dec 10; 12 (12): CD014439CD014439.
AbstractThis 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.Copyright © 2024 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd.
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