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Amicus Therapeutics, Inc. — Rare Disease Pipeline | Kisho | Kisho

Amicus Therapeutics, Inc.

Other

4

Orphan Designations

0

FDA Approvals

16

Rare Diseases

1

News Articles

Disease	Drug(s)	Designation	Approved
Fabry disease	GALAFOLD	Orphan Designation	-
Gaucher disease	isofagomine tartrate	Orphan Designation	-
adult embryonal tumor with multilayered rosettes, c19mc-altered	GALAFOLD	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	Pombiliti and Opfolda	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	Pombiliti and Opfolda	Orphan Designation	-
adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy	GALAFOLD	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	Pombiliti and Opfolda	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	Pombiliti and Opfolda	Orphan Designation	-
cataract 13 with adult I phenotype	GALAFOLD	Orphan Designation	-
gallbladder papillary neoplasm with an associated invasive carcinoma	GALAFOLD	Orphan Designation	-
glycogen storage disease II	Pombiliti and Opfoldaduvoglustat hydrochloride	Orphan Designation	-
has an isolated presentation	GALAFOLD	Orphan Designation	-
nut midline carcinoma	GALAFOLD	Orphan Designation	-
polydactyly of an index finger	GALAFOLD	Orphan Designation	-
polydactyly of an index finger, bilateral	GALAFOLD	Orphan Designation	-
primary adult heart tumor	Pombiliti and Opfolda	Orphan Designation	-

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