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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 |
---|---|---|---|---|
Ascorbic acid | Facioscapulohumeral muscular dystrophy | Université de Montpellier | November 18, 2016 | |
Vaccine consisting of 5 survivin peptides with different human leukocyte antigen restrictions | Ovarian cancer | Dr Ulrich Granzer | November 18, 2016 | |
Budesonide | IgA Nephropathy | Pharmalink AB | November 18, 2016 | |
Ibrutinib | Graft Versus Host Disease | Janssen-Cilag International NV | November 18, 2016 | |
L-selenomethionine | Facioscapulohumeral muscular dystrophy | Université de Montpellier | November 18, 2016 | |
Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Arg-Gly-Gly-Asp-Leu-Leu-Pro-Arg-Gly-Ser | Huntington disease | Dr Ulrich Granzer | November 18, 2016 | |
N-(5-(6-chloro-2,2-difluorobenzo[d][1,3]dioxol-5-yl)pyrazin-2-yl)-2-fluoro-6-methylbenzamide | Acute pancreatitis | Cogas Pharma Limited | November 18, 2016 | |
Particles comprised of methacrylic acid based co-polymer, cross-linked with a bi-functional cross-linker, purified to bind L-phenylalanine and L-phenylalanine containing peptides | Hyperphenylalaninaemia | MipSalus ApS | November 18, 2016 | |
R-azasetron besylate | Sensorineural hearing loss | Sensorion | November 18, 2016 | |
Recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein | Aromatic amino acid decarboxylase deficiency | Diamond BioPharm Limited | November 18, 2016 | |
Sodium benzoate | N-acetylglutamate synthetase deficiency | Lucane Pharma SA | November 18, 2016 | |
Valproic acid | Diffuse Large B-cell Lymphoma | Valcuria AB | November 18, 2016 | |
2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde | Sickle cell anemia | SynteractHCR Deutschland GmbH | November 18, 2016 | |
Zinc gluconate | Facioscapulohumeral muscular dystrophy | Université de Montpellier | November 18, 2016 | |
5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride | X-linked adrenoleukodystrophy | Minoryx Therapeutics S.L. | November 18, 2016 | |
Brincidofovir | Smallpox | Chimerix UK Ltd | November 18, 2016 | |
Adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene | Glycogen storage disease type Ia | Pharma Gateway AB | November 18, 2016 | |
Human monoclonal antibody against activin A | Fibrodysplasia ossificans progressiva | Regeneron Ireland | November 18, 2016 | |
Allogeneic cytomegalovirus-specific cytotoxic T lymphocytes | Cytomegalovirus infection in patients with impaired cell-mediated immunity | Atara Biotherapeutics Ireland Limited | November 18, 2016 | |
Live-attenuated non-replicativePseudomonas aeruginosastrain expressing large T antigen of Merkel cell polyomavirus | Merkel cell cancer | APCure SAS | November 18, 2016 | |
Allogeneic peripheral blood mononuclear cells incubated ex vivo with 16, 16-dimethyl prostaglandin E2 and dexamethasone | Treatment in haematopoietic stem cell transplantation | Fate Therapeutics Ltd | November 18, 2016 | |
Synthetic human hepcidin | Sickle cell anemia | Cogas Pharma Limited | November 18, 2016 | |
Alpha-tocopherol | Facioscapulohumeral muscular dystrophy | Université de Montpellier | November 18, 2016 | |
Diazepam buccal soluble film | Selected, refractory patients with epilepsy who are on stable regimens of antiepileptic drugs (AED) and who require intermittent use of diazepam to control bouts of increased seizure activity (acute repetitive seizures) | Aquestive Therapeutics;30 Technology Drive South;Warren, New Jersey, 07059 | November 10, 2016 | |
Sargramostim | Acute Radiation Syndrome Acute radiation syndrome | Sanofi Genzyme;500 Kendall Street;Cambridge, Massachusetts, 02142 | November 9, 2016 |