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Treatments
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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Country | Product | Indication | Sponsor | Date |
---|---|---|---|---|
3-[3-(6-guanidino-1-oxoisoindolin-2yl) propanamido]-3-(pyridine-3yl) propanoic acid dihydrochloride | Retinopathy of prematurity | Advanced Imaging Projects, LLC;7947 Brookside Ct.;Lake Worth, Florida, 33467 | January 17, 2017 | |
Trans-resveratrol | Spinocerebellar ataxia | Luis Pereira de Almeida | January 12, 2017 | |
Exon 53 specific antisense morpholino oligonucleotide. | Duchenne muscular dystrophy | NS Pharma, Inc.;140 East Ridgewood Avenue, Suite 280S;Paramus, New Jersey, 07652 | January 12, 2017 | |
(E)-N’-(1-(5-Chloro-2-hydroxy phenyl)Ethylidene)-3-((4-Methyl Piperazin-1-yl) Sulfonyl) Benzohydrazide Mesylate | Ewing's sarcoma | Salarius Pharmaceuticals;2450 Holcombe Boulevard, Suite J-608;Houston, Texas, 77021 | January 12, 2017 | |
(E)-N�-(1-(5-Chloro-2-hydroxy phenyl)Ethylidene)-3-((4-Methyl Piperazin-1-yl) Sulfonyl) Benzohydrazide Mesylate | Ewing's sarcoma | Salarius Pharmaceuticals;2450 Holcombe Boulevard, Suite J-608;Houston, Texas, 77021 | January 12, 2017 | |
[5,10,15,20-tetrakis(4-carboxyphenyl)-21H,23H-porphine]manganese(III) chloride | Cockayne syndrome | Institut Pasteur | January 12, 2017 | |
3-pentylbenzeneacetic acid sodium salt | Alström syndrome | ProMetic Pharma SMT Limited | January 12, 2017 | |
Antroquinonol | Pancreatic Cancer | Biological Consulting Europe Ltd | January 12, 2017 | |
Autologous dendritic cells incubated ex vivo with zebularine and factor VIII | Haemophilia A | Idogen AB | January 12, 2017 | |
Anti-interleukin 21 (NNC0114-0006) in combination with liraglutide | Type I Diabetes Mellitus with residual beta cell function | Novo Nordisk, Inc.;800 Scudders Mill Road, P. O. Box 846;New Jersey, 08536 | January 12, 2017 | |
(6aR, 10aR)-3-(1′,1′-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid | Systemic scleroderma | TMC Pharma Services Ltd | January 12, 2017 | |
Doxorubicin hydrochloride in a lipid-based pegylated nanoparticle modified with a 31-aminoacid peptide targeting nucleolin | Malignant pleural mesothelioma | TREAT U, S.A. | January 12, 2017 | |
5-aminolevulinic acid | Glioma | Centre Hospitalier Universitaire de Lille | January 12, 2017 | |
Human donor haematopoietic stem and progenitor cells that have been treated ex vivo with the protein transduction domain of the HIV-1 transactivation protein fused to MYC transcription factor | Treatment in haematopoietic stem cell transplantation | Coté Orphan Consulting UK Limited | January 12, 2017 | |
Fluticasone propionate | Eosinophilic esophagitis Eosinophilic esophagitis | Adare Pharmaceuticals srl | January 12, 2017 | |
Human hepatoma cell line HepaRG in bioartificial liver | Acute liver failure | Hep-Art Medical Devices BV | January 12, 2017 | |
Genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimized shRNA-insensitive wildtype PABPN1 | Oculopharyngeal muscular dystrophy | Clinipace GmbH | January 12, 2017 | |
Leuprorelin acetate | Congenital hypogonadotropic hypogonadism | Stichting Centre for Human Drug Research (CHDR) | January 12, 2017 | |
Humanised IgG1 monoclonal antibody against the receptor-binding site of human placental growth factor | Medulloblastoma | Oncurious NV | January 12, 2017 | |
Glyburide | Severe cerebral edema in patients with acute ischemic stroke | Biogen Chesapeake LLC;225 Binney Street;Cambridge, Massachusetts, 02142 | January 12, 2017 | |
Pioglitazone hydrochloride | Sensorineural hearing loss | Regiomedica GmbH | January 12, 2017 | |
Tadekinig alfa | Hemophagocytic lymphohistiocytosis | AB2 Bio Ltd;EPFL Innovation Park, Building B;Lausanne | January 12, 2017 | |
Hydroxychloroquine | Antiphospholipid syndrome | Centre Hospitalier Universitaire d'Angers | January 12, 2017 | |
Methyldopa | Type 1 diabetes in DQ8 positive patients with residual beta cell function | IM Therapeutics LLC;11001 West 120th Avenue, Suite 400;Broomfield, Colorado, 80021 | January 12, 2017 | |
Recombinant adeno-associated viral vector serotype 9 containing the human N-alpha-acetylglucosaminidase gene | Mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) | Abeona Therapeutics Europe SL | January 12, 2017 |