Effect of creatine monohydrate on motor function in children with facioscapulohumeral muscular dystrophy: A multicenter, randomized, double‐blind placebo‐controlled crossover trial

CrM is safe and well tolerated in children with FSHD. Although CrM had no effect on motor function as measured by the MFM-32 compared with placebo, there were trends toward improvement in the 6-minute walk distance and other secondary outcome measures. This study confirms the feasibility of conducting clinical trials in children with FSHD. Further assessment of the efficacy of CrM in pediatric FSHD is warranted in a larger randomized controlled clinical trial. Future studies may benefit from stratifying population cohorts according to functional ability or by MRI fat infiltration measurements.

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Publication Details

Journal
Pharmacotherapy The Journal of Human Pharmacology and Drug Therapy
Published
2025-05-14
DOI
https://doi.org/10.1002/phar.70025
Citations
2
Primary Topic
Muscle Physiology and Disorders
Type
article
Field-Weighted Citation Impact
1.61

Funders

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article

Effect of creatine monohydrate on motor function in children with facioscapulohumeral muscular dystrophy: A multicenter, randomized, double‐blind placebo‐controlled crossover trial

Eppie M. Yiu, Nuran Dilek, Zoe E. Davidson, Monique M. Ryan et al.
2 citations
Pharmacotherapy The Journal of Human Pharmacology and Drug Therapy
Muscle Physiology and Disorders
1.61
article

Effect of creatine monohydrate on motor function in children with facioscapulohumeral muscular dystrophy: A multicenter, randomized, double‐blind placebo‐controlled crossover trial

Eppie M. Yiu, Nuran Dilek, Zoe E. Davidson, Monique M. Ryan, Chad Heatwole, Martin Bruce Delatycki, Katy de Valle, Anneke Grobler, Anita Cairns, Nisha Varma, Kate Carroll, Ian R. Woodcock, David Metz, Michael Kean
article en
2 citations

Abstract

CrM is safe and well tolerated in children with FSHD. Although CrM had no effect on motor function as measured by the MFM-32 compared with placebo, there were trends toward improvement in the 6-minute walk distance and other secondary outcome measures. This study confirms the feasibility of conducting clinical trials in children with FSHD. Further assessment of the efficacy of CrM in pediatric FSHD is warranted in a larger randomized controlled clinical trial. Future studies may benefit from stratifying population cohorts according to functional ability or by MRI fat infiltration measurements.

Pharmacotherapy The Journal of Human Pharmacology and Drug TherapyVol. 45(6)
Royal Children's Hospital (AU), The University of Melbourne (AU), Victorian Clinical Genetics Services (AU), Murdoch Children's Research Institute (AU), Children's Health Queensland Hospital and Health Service (AU), University of Rochester (US), Monash University (AU)
FSHD Global Research Foundation
Good health and well-being
Openalex Percentile: Top 17%
Muscle Physiology and Disorders
1.61
DOIOpen Access PDFOpenAlex
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