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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
C-type natriuretic peptide conjugated to a multi-arm polyethylene glycol carrier molecule through a cleavable linker Achondroplasia Feb 27, 2019
autologous CD34+ Cells Enriched with Blood Derived Mitochondria Pearson Syndrome Feb 26, 2019
recombinant botulinum vaccine A/B Prevention of botulism caused by inhalational intoxication with botulinum neurotoxin (BoNT) serotype A, subtype A1, and BoNT serotype B, subtype B1 Feb 26, 2019
dexamethasone Multiple myeloma Feb 26, 2019
Risdiplam Spinal muscular atrophy Feb 26, 2019
Losartan Epidermolysis bullosa Feb 26, 2019
Lentiviral vector encoding human coagulation factor IX Haemophilia B Feb 26, 2019
Humanised IGg1 monoclonal antibody targeting human transferrin receptor conjugated to human iduronate-2-sulfatase Mucopolysaccharidosis II (Hunter’s syndrome) Feb 26, 2019
Autologous adult live cultured osteoblasts Non-traumatic osteonecrosis Feb 26, 2019
Allogeneic cultured postnatal thymus-derived tissue Severe combined immunodeficiency due to FOXN1 deficiency Feb 26, 2019
Allogeneic cultured postnatal thymus-derived tissue CHARGE syndrome Feb 26, 2019
Allogeneic cultured postnatal thymus-derived tissue DiGeorge syndrome Feb 26, 2019
9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester Infantile neuroaxonal dystrophy Feb 26, 2019
Poly(N-acetyl, N-arginyl)glucosamine Cystic fibrosis Feb 26, 2019
6-{[(1R,2S)-2-aminocyclohexyl]amino-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridin-3-one monocitrate Follicular lymphoma Feb 25, 2019
5-(4-((2S,5S)-5-(4-chlorobenzyl)-2-methylmorpholino)piperidin-1-yl)-1H-1,2,4-triazol-3-amine Idiopathic pulmonary fibrosis Feb 25, 2019
enzyme transport vehicle specific for the transferrin receptor fused to iduronate 2-sulfatase Mucopolysaccharidosis type II Feb 19, 2019
verdiperstat Multiple system atrophy (MSA) Feb 15, 2019
recombinant adeno-associated viral vector serotype S3 containing codon-optimized expression cassette encoding human coagulation factor IX variant Hemophilia B Feb 15, 2019
A small molecule inhibitor of the pro-survival protein Myeloid Cell Leukemia sequence 1 (MCL 1) Acute Myeloid Leukemia Feb 15, 2019