Fabry Disease

Amicus Therapeutics announces European regulatory status on Amigal for Fabry disease

Published in Gene Therapy Weekly, May 11th, 2006

Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA) has recommended orphan medicinal product designation for Amigal (migalastat hydrochloride or AT1001).

Amigal is being developed as an oral therapy for the treatment of Fabry disease, a lysosomal storage disorder caused by an enzyme deficiency that leads to pain, kidney failure, and an increased risk of heart attack and stroke. Amigal is currently being studied in Phase II clinical trials in the U.S.,...

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Source: Gene Therapy Weekly (2006-05-11)

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