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N-of-1 trial of salbutamol in hyperkalaemic periodic paralysis

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  • Contributors GJvdW, BGMvE and BCS obtained funding for the study and designed the study with input of ECM, JR, CGJS, RW, BvV, HG, RCG and JS. BCS is the primary investigator and responsible for data collection. BvV is responsible for the randomisation process and production of blinded study medication. HMG and BCS are responsible for the data analysis. CGJS and BCS are responsible for the EMG measurements. JR, CGJS and BGMvE supervised BCS during the study. BCS wrote the first draft of the manuscript. All authors have read, critically revised and approved the final version of the manuscript.

  • Funding This study is funded by ZonMw, The Netherlands Organisation for Health Research and Development (ZonMw project number, 152002029). The funder had no role in the design and conduct of the study; collection, management, analysis and interpretation of the data; preparation, review or approval of the manuscript; and decision to submit the manuscript for publication.The first author (BCS) and trial statistician (HG) had full access to all the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis. Several authors of this publication (BSC, CGJS, RW, BGMvE and JR) are members of the European Reference Network for Neuromuscular Diseases (EURO-NMD).

  • Competing interests Dr RG reports grants (during the conduct of the study) from NIH, The Muscular dystrophy association and The Parent Project for Muscular dystrophy, and, personal fees from Strongbridge Pharmaceuticals, Sarepta Pharmaceuticals, Marathon Pharmaceuticals, Stealth Pharmaceuticals, outside the submitted work; Dr JS reports grants from NINDS, grants from FSH Society, personal fees from Acceleron, personal fees from Sarepta, personal fees from Fulcrum, personal fees from PTC, personal fees from Dyne Therapeutics, outside the submitted work; Dr BGMvE reports grants from European Union’s Horizon 2020 research and innovation programme (Murab), grants from European FP7 programme (OPTIMISTIC), grants from Association Francaise contre les Myopathies, grants from Global FSH, grants from The Netherlands Organisation for Health Research and Development (ZonMw), grants from Prinses Beatrix Spierfonds, grants from Stiching Spieren voor Spieren, grants from Dutch FSHD Foundation, grants from Netherlands Organisation for Scientific Research (NWO), personal fees from Fulcrum, outside the submitted work. All other authors declare that they have no financial or non-financial competing interests.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.