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migalastat (Galafold®)


Status: In progress

Long-term treatment of  adolescents aged 12-15 years with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation.

Medicine details

Medicine name migalastat (Galafold®)
Formulation 123 mg capsule
Reference number 4268

As above

Company Amicus Therapeutics UK Ltd
BNF chapter Nutrition & blood
Submission type Licence extension for paediatric use
Status In progress
AWMSG meeting date 18/10/2022
Date of issue TBA
Commercial arrangement PAS
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