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Treatment Database

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

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Product Indication Date Designated
Double stranded oligomer ARO-APOC3 RNA interference-based liver targeted therapeutic Familial Chylomicronemia Syndrome Jun 20, 2019
Balipodect CDKL5 deficiency disorder Jun 19, 2019
L-Leucyl-L-prolyl-L-prolyl-L-leucyl-L-prolyl-L-tyrosyl-L-proline, acetate salt Amyotrophic Lateral Sclerosis Jun 19, 2019
2-(2-{[2-(1H-benzimidazol-2-yl)ethyl]amino}ethyl)-N-[(3-fluoropyridin-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride Beta-thalassemia Jun 17, 2019
Balipodect Fragile X Syndrome Jun 13, 2019
adoptive, anti-FLT3 chimeric antigen receptor T cell therapy generated through transduction of autologous T-cells with a self-inactivating human immunodeficiency virus-1 based lentiviral vector Acute myeloid leukemia Jun 13, 2019
parsaclisib Mantle cell lymphoma Jun 12, 2019
4-((L-valyl)oxy)butanoic acid Narcolepsy Jun 12, 2019
Venetoclax Myelodysplastic syndromes Jun 12, 2019
Autologous CD34+ hematopoietic stem and progenitor cells transfected with zinc finger nuclease messenger RNAs SB-mRENH1 and SB-mRENH2 Sickle Cell Disease Jun 11, 2019
humanized anti CD20 monoclonal antibody Dermatomyositis Jun 11, 2019
fosgemcitabine palabenamide Biliary tract cancer Jun 11, 2019
adeno-associated virus serotype rh10 encoding artificial anti-SOD1 miRNA Amyotrophic Lateral Sclerosis Jun 11, 2019
72 amino acid proprietary recombinant protein antagonist for the CXCR1 and CXCR2 receptors of interleukin-8 Pancreatic Cancer Jun 04, 2019
flubrobenguane (18F) Diagnostic for the management of neuroendocrine tumors Jun 04, 2019
dornase alfa Sarcoidosis Jun 04, 2019
casimersen Duchenne muscular dystrophy Jun 04, 2019
bardoxolone methyl Autosomal dominant polycystic kidney disease Jun 03, 2019
4-Methyl-3-quinolin-3-ylethynylbenzoic acid N'-(2-chloro-6- methylbenzoyl) hydrazide Chronic myeloid leukemia Jun 03, 2019
2-(4-oxo-3-((5-(trifluoromethyl)benzo[d]thiazol-2-yl)methyl)-3,4-dihydrothieno[3,4-d]pyridazin-1-yl)acetic acid Galactosemia May 23, 2019