Long-term Natural History of Pediatric Dominant and Recessive RYR1 Related Myopathy.

Anna SarkozyMario SaDeborah RidoutMiguel Angel Fernandez-GarciaMaria Grazia DistefanoMarion MainJennie SheehanAdnan Y ManzurPinki MunotStephanie RobbElizabeth WraigeRos QuinlivanMariacristina ScotoGiovanni BaranelloVasantha GowdaRachael MeinRahul PhadkeHeinz JungbluthFrancesco Muntoni

Published in: Neurology (2023)

-related myopathies that may inform management and provide essential milestones for future therapeutic interventions.

Keyphrases

physical activity
intellectual disability
current status