For help on how to get the results you want, see our search tips.
1795 results
-
List item
Orphan designation: Heterologous human adult liver-derived progenitor cells for: Treatment of argininosuccinic aciduria
Date of designation: 17/07/2013, Positive, -
List item
Orphan designation: Masitinib mesilate for: Treatment of pancreatic cancer
Date of designation: 28/10/2009, Positive, -
List item
Orphan designation: Recombinant protein derived from the saliva of the Ornithodoros moubata tick (nomacopan) for: Treatment of Guillain-Barré syndrome
Date of designation: 27/06/2016, Positive, Last updated: 26/07/2016 -
List item
Orphan designation: 2',3',5'-Tri-O-acetyluridine for: Treatment of 5-fluorouracil overdose
Date of designation: 15/05/2009, Positive, Last updated: 09/06/2009 -
List item
Orphan designation: 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid for: Treatment of systemic sclerosis
Date of designation: 12/03/2013, Positive, Last updated: 18/09/2020 -
List item
Orphan designation: Eflornithine for: Treatment of familial adenomatous polyposis (FAP)
Date of designation: 20/09/2010, Positive, Last updated: 29/04/2019 -
List item
Orphan designation: Propranolol for: Treatment of soft tissue sarcoma
Date of designation: 12/12/2016, Positive, -
List item
Orphan designation: Mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene for: Treatment of Usher syndrome
Date of designation: 04/07/2014, Positive, -
List item
Orphan designation: Repertaxin L-lysine salt for: Prevention of delayed graft function in organ transplant
Date of designation: 19/09/2001, Positive, -
List item
Orphan designation: Ursodeoxycholic acid for: Treatment of Niemann-Pick disease
Date of designation: 22/05/2017, Positive, Last updated: 25/04/2019 -
List item
Orphan designation: Pyridoxal 5'-phosphate for: Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Date of designation: 15/10/2014, Positive, -
List item
Orphan designation: Recombinant human histone H1.3 and recombinant human N-bis-met-histone H1.3 for: Treatment of acute myeloid leukaemia
Date of designation: 20/12/2007, Positive, Last updated: 13/03/2013 -
List item
Orphan designation: Methylthioninium for: treatment of frontotemporal dementia with parkinsonism-17
Date of designation: 26/11/2010, Positive, Last updated: 28/03/2019 -
List item
Orphan designation: Poly-cyclodextrin-bis-cysteine-PEG3400-camptothecin-conjugate for: Treatment of ovarian cancer
Date of designation: 20/03/2017, Positive, Last updated: 05/05/2017 -
List item
Orphan designation: Human MHC non-restricted cytotoxic T-cell line for: Treatment of ovarian cancer
Date of designation: 30/11/2009, Positive, -
List item
Orphan designation: afamelanotide for: Treatment of solar urticaria
Date of designation: 24/07/2009, Positive, Last updated: 11/04/2016 -
List item
Orphan designation: Adenovirus associated viral vector serotype 4 containing the human RPE65 gene for: Treatment of Leber's congenital amaurosis
Date of designation: 22/10/2007, Positive, -
List item
Orphan designation: Nitric oxide for: Treatment of cystic fibrosis
Date of designation: 15/10/2014, Positive, -
List item
Orphan designation: 68Ga-DOTA-pABzA-DIG-dPhe-Gln-Trp-Ala-Val-Gly-His-NHCH[(CH2-CH(CH3)2]2 for: Diagnosis of gastrointestinal stromal tumours
Date of designation: 12/12/2016, Positive, -
List item
Orphan designation: N'-(5-chloro-2-hydroxy-3-methylbenzylidene)-2,4-dihydroxybenzhydrazide for: Treatment of partial deep dermal and full-thickness burns
Date of designation: 22/09/2008, Positive, Last updated: 06/04/2011 -
List item
Orphan designation: Veliparib for: Treatment of ovarian cancer
Date of designation: 17/12/2010, Positive, Last updated: 29/05/2018 -
List item
Orphan designation: Cladribine for: Treatment of indolent non-Hodgkin's lymphoma
Date of designation: 18/09/2001, Expired, Last updated: 18/06/2014 -
List item
Orphan designation: Volasertib for: Treatment of acute myeloid leukaemia
Date of designation: 26/03/2014, Positive, -
List item
Orphan designation: 5,6,7,8-Tetrahydrobiopterin for: Treatment of hyperphenylalaninaemia
Date of designation: 02/10/2003, Positive, Last updated: 23/02/2007 -
List item
Orphan designation: Caffeine citrate for: Prevention of bronchopulmonary dysplasia
Date of designation: 11/04/2014, Positive,