Oral N-acetylcysteine Trial for RYR1-related Myopathies

Authors

  • Vamshi Rao Division of Neurology, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL; Departments of Pediatrics and Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL

DOI:

https://doi.org/10.15844/pedneurbriefs-36-4

Keywords:

RYR1-RM, N-acteylcysteine, Clinical trial

Abstract

Investigators from NIH, Hyperion Biotechnology Inc., and Hospital for Sick Children studied the effect of oral N-acetylcysteine (NAC) on decreasing oxidative stress and increasing physical endurance in individuals with ryanodine receptor 1-related myopathies (RYR1-RM).

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Published

2022-12-30

Issue

Section

Neuromuscular Disorders