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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
tiratricol Monocarboxylate 8 Transporter (MCT8) Deficiency (Allan-Herndon-Dudley-Syndrome) Jan 16, 2019
adeno-associated viral vector 9-containing the human heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT) gene (AAV9- HGSNAT) Sanfilippo Syndrome IIIC (also known as Mucopolysaccharidosis IIIC/MPS IIIC) Jan 16, 2019
ribitol Limb-girdle muscular dystrophy type 2I Jan 16, 2019
odevixibat Biliary Atresia Jan 16, 2019
N-(bromoacetyl)-3,3-dinitroazetidine Glioblastoma multiforme Jan 16, 2019
D-Galactose Phosphoglucomutase 1 Deficiency Jan 14, 2019
cusatuzumab Acute myeloid leukemia Jan 14, 2019
Autotaxin inhibitor Idiopathic Pulmonary Fibrosis Jan 14, 2019
2-(5-(2-((2,3-dihydro-1H-inden-2-yl)amino)pyrimidin-5-yl)-1,3,4-oxadiazol-2-yl)-1-(1,4,6,7-tetrahydro-5H-[1,2,3]triazolo[4,5-c]pyridin-5-yl)ethan-1-one Idiopathic Pulmonary Fibrosis Jan 14, 2019
Rozanolixizumab Immune thrombocytopenia Jan 11, 2019
Mercaptamine-pantetheine disulfide Rett syndrome Jan 11, 2019
Human glucagon-like peptide-2 analogue linked to a human immunoglobulin Fc fragment Short bowel syndrome Jan 11, 2019
Acetylleucine Ataxia telangiectasia Jan 11, 2019
bromelain + acetylcysteine Pseudomyxoma peritonei with Bromelain and Acetylcysteine Dec 27, 2018
apraglutide Short bowel syndrome (SBS) Dec 20, 2018
Autologous adult live cultured osteoblasts Osteonecrosis Dec 20, 2018
iberdomide Multiple myeloma Dec 19, 2018
PEGylated peptide inhibitor of complement C3 C3 glomerulopathy Dec 19, 2018
Synthetic PreImplantation Factor Acute Radiation Syndrome Dec 19, 2018
small interfering RNA agent that targets human transforming growth factor-�1 messenger RNA Idiopathic pulmonary fibrosis Dec 19, 2018