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Stealth Biotherapeutics Inc. — Rare Disease Pipeline | Kisho | Kisho

Stealth Biotherapeutics Inc.

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Barth syndrome	Forzinity	Orphan Designation	-
Duchenne muscular dystrophy	Elamipretide HCl	Orphan Designation	-
Friedreich ataxia	elamipretide	Orphan Designation	-
Friedreich ataxia 1	elamipretide	Orphan Designation	-
Friedreich ataxia 2	elamipretide	Orphan Designation	-
Kearns-Sayre syndrome	elamipretide	Orphan Designation	-
Leber hereditary optic neuropathy	elamipretide	Orphan Designation	-
Leber hereditary optic neuropathy, autosomal recessive	elamipretide	Orphan Designation	-
Leber optic atrophy and dystonia	elamipretide	Orphan Designation	-
amyotrophic lateral sclerosis	(R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, tris hydrochloride	Orphan Designation	-
hypotonia-failure to thrive-microcephaly syndrome	Forzinity	Orphan Designation	-
insensitivity to pain with hyperplastic Myelinopathy	Forzinity	Orphan Designation	-
mitochondrial myopathy-cerebellar ataxia-pigmentary retinopathy syndrome	elamipretide	Orphan Designation	-
neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome	elamipretide	Orphan Designation	-
pediatric acute-onset neuropsychiatric syndrome	Forzinity	Orphan Designation	-
pediatric hepatocellular carcinoma	Forzinity	Orphan Designation	-
pediatric ovarian dysgerminoma	Forzinity	Orphan Designation	-
pediatric ovarian germ cell tumor	Forzinity	Orphan Designation	-
pediatric-onset Graves disease	Forzinity	Orphan Designation	-
twin to twin transfusion syndrome	Forzinity	Orphan Designation	-

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