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Actelion Pharmaceuticals Ltd — Rare Disease Pipeline | Kisho | Kisho

Actelion Pharmaceuticals Ltd

Other

8

Orphan Designations

0

FDA Approvals

16

Rare Diseases

0

News Articles

Disease	Drug(s)	Designation	Approved
Clostridium difficile colitis	cadazolid	Orphan Designation	-
Gorham-Stout disease	bosentan	Orphan Designation	-
absence of the pulmonary artery	Uptravi	Orphan Designation	-
arteriovenous malformations of the brain	Uptravi	Orphan Designation	-
developmental and epileptic encephalopathy, 7	Opsumit	Orphan Designation	-
fibrous dysplasia	bosentan	Orphan Designation	-
heritable pulmonary arterial hypertension	UptraviOpsumitTracleer	Orphan Designation	-
idiopathic pulmonary arterial hypertension	UptraviOpsumitTracleer	Orphan Designation	-
idiopathic pulmonary fibrosis	macitentanbosentan	Orphan Designation	-
medial condensing osteitis of the clavicle	bosentan	Orphan Designation	-
nephropathy, progressive, with deafness	Opsumit	Orphan Designation	-
pili torti-developmental delay-neurological abnormalities syndrome	miglustat	Orphan Designation	-
pulmonary arterial hypertension	UptraviOpsumitTracleer	Orphan Designation	-
pulmonary hypertension, primary, 1	UptraviOpsumitTracleer	Orphan Designation	-
pulmonary hypertension, primary, 2	UptraviOpsumitTracleer	Orphan Designation	-
ravine syndrome	miglustat	Orphan Designation	-

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