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Orphan Europe SARL — Rare Disease Pipeline | Kisho | Kisho

Orphan Europe SARL

Other

4

Orphan Designations

0

FDA Approvals

17

Rare Diseases

0

News Articles

Disease	Drug(s)	Designation	Approved
3-methylcrotonyl-CoA carboxylase 1 deficiency	Carbaglu	Orphan Designation	-
Angelman syndrome	Carbaglu	Orphan Designation	-
acyl-CoA dehydrogenase 9 deficiency	Carbaglu	Orphan Designation	-
aortic valve stenosis	Carbaglu	Orphan Designation	-
homocystinuria	Cystadane	Orphan Designation	-
hyperammonemia due to N-acetylglutamate synthase deficiency	Carbaglu	Orphan Designation	-
intellectual developmental disorder, autosomal recessive 83	Carbaglu	Orphan Designation	-
methylmalonic acidemia with homocystinuria, type cblJ	Cystadane	Orphan Designation	-
methylmalonic aciduria and homocystinuria type cblC	Cystadane	Orphan Designation	-
methylmalonic aciduria and homocystinuria type cblF	Cystadane	Orphan Designation	-
mitochondrial DNA depletion syndrome 6 (hepatocerebral type)	Carbaglu	Orphan Designation	-
myelodysplastic syndrome	Heme arginate	Orphan Designation	-
recurrent metabolic encephalomyopathic crises-rhabdomyolysis-cardiac arrhythmia-intellectual disability syndrome	Carbaglu	Orphan Designation	-
symptomatic form of Coffin-Lowry syndrome in female carriers	Heme arginate	Orphan Designation	-
symptomatic form of fragile X syndrome in female carrier	Heme arginate	Orphan Designation	-
symptomatic form of hemophilia A in female carriers	Heme arginate	Orphan Designation	-
systemic primary carnitine deficiency disease	Carbaglu	Orphan Designation	-

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