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Hoechst Marion Roussel

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
ACTB-associated syndromic thrombocytopenia	Refluden	Orphan Designation	-
Chagas cardiomyopathy	Ornidyl	Orphan Designation	-
Mycobacterium avium complex disease	Rifapentine	Orphan Designation	-
SATB2 associated disorder	Refluden	Orphan Designation	-
Sjogren syndrome	Rifapentine	Orphan Designation	-
anosmia for isobutyric acid	RefludenRifadin I.V.	Orphan Designation	-
apolipoprotein A-II amyloidosis	Rifapentine	Orphan Designation	-
ataxia telangiectasia	Rifapentine	Orphan Designation	-
double nail for fifth toe	RefludenRifadin I.V.	Orphan Designation	-
hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain	Rifadin I.V.	Orphan Designation	-
hemophilia A	Rifapentine	Orphan Designation	-
heparin-induced thrombocytopenia	Refluden	Orphan Designation	-
idiopathic copper-associated cirrhosis	Refluden	Orphan Designation	-
infection by Trypanosoma gambiense	Ornidyl	Orphan Designation	-
infection by Trypanosoma rhodesiense	Ornidyl	Orphan Designation	-
modifier, X-linked, for Neurofunctional defects	Refluden	Orphan Designation	-
proteasome-associated autoinflammatory syndrome 1	Refluden	Orphan Designation	-
proteasome-associated autoinflammatory syndrome 3	Refluden	Orphan Designation	-
pulmonary tuberculosis	Priftin	Orphan Designation	-
spatial visualization, aptitude for	Refluden	Orphan Designation	-
triple-A syndrome	Rifapentine	Orphan Designation	-
tuberculosis	Rifater	Orphan Designation	-