Migalastat Hydrochloride Dispersible Tablet clinical trials

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12-month Treatment with Migalastat in Pediatric Subjects (aged 2 to < 12 years) with Fabry Disease and Amenable GLA Variants

This study looks at a medicine called migalastat for children aged 2 to under 12 with Fabry disease. It checks how safe and effective the medicine is over 12 months, focusing on how the body uses it and if it helps with Fabry symptoms.