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Insmed Incorporated — Rare Disease Pipeline | Kisho | Kisho

Insmed Incorporated

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
COG4-congenital disorder of glycosylation	Iplex	Orphan Designation	-
Kluver-Bucy syndrome	rhIGF-I/rhIGFBP-3	Orphan Designation	-
Laron syndrome	Iplex	Orphan Designation	-
Mycobacterium avium complex disease	Arikayce	Orphan Designation	-
amyloidosis bronchopulmonary	liposomal amikacin	Orphan Designation	-
bronchiectasis with or without elevated sweat chloride 2	liposomal amikacin	Orphan Designation	-
bronchiectasis with or without elevated sweat chloride 3	liposomal amikacin	Orphan Designation	-
bronchopulmonary dysplasia	liposomal amikacin	Orphan Designation	-
chlamydiaceae infections	Arikayce	Orphan Designation	-
cystic fibrosis	liposomal amikacin	Orphan Designation	-
deltaretrovirus infections	Arikayce	Orphan Designation	-
gingival fibromatosis-hypertrichosis syndrome	rhIGF-I/rhIGFBP-3	Orphan Designation	-
growth hormone insensitivity syndrome	Iplex	Orphan Designation	-
growth hormone insensitivity syndrome with immune dysregulation	Iplex	Orphan Designation	-
inflammatory bowel disease 1	Iplex	Orphan Designation	-
major affective disorder 3	recombinant human insulin-like growth factor-I/insulin-like growth factor binding protein-3	Orphan Designation	-
major salivary gland carcinoma ex pleomorphic adenoma	recombinant human insulin-like growth factor-I/insulin-like growth factor binding protein-3	Orphan Designation	-
major salivary gland mucoepidermoid carcinoma	recombinant human insulin-like growth factor-I/insulin-like growth factor binding protein-3	Orphan Designation	-
myotonic dystrophy	mecasermin	Orphan Designation	-
paroxysmal extreme pain disorder	rhIGF-I/rhIGFBP-3	Orphan Designation	-
primary ciliary dyskinesia 1	liposomal amikacin	Orphan Designation	-
severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome	Iplex	Orphan Designation	-
spirochaetales infections	Arikayce	Orphan Designation	-

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