For help on how to get the results you want, see our search tips.
11 results
Categories
Human Remove Human filter
-
List item
Orphan designation: dimethyl fumarate for: Treatment of adrenoleukodystrophy
Date of designation: 09/01/2020, Positive, Last updated: 11/11/2022 -
List item
Orphan designation: 2-(3,7-Dimethyl-octa-2, 6-dienyl)-6-ethylamino-3-hydroxy-5-pentyl-[1,4]benzoquinone for: Treatment of Huntington's disease
Date of designation: 09/01/2020, Positive, Last updated: 15/05/2020 -
List item
Orphan designation: adeno-associated virus serotype 9 vector containing human n-acetylgalactosamine-6-sulfate sulfatase gene for: Treatment of mucopolysaccharidosis type IVA (Morquio A Syndrome)
Date of designation: 09/01/2020, Positive, Last updated: 08/07/2022 -
List item
Orphan designation: tripotassium citrate monohydrate, Potassium hydrogen carbonate for: Treatment of cystinuria
Date of designation: 09/01/2020, Positive, Last updated: 13/04/2023 -
List item
Orphan designation: nicardipine for: Treatment of non-traumatic subarachnoid haemorrhage
Date of designation: 09/01/2020, Positive, Last updated: 19/05/2020 -
List item
Orphan designation: adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA for: Treatment of Fabry disease
Date of designation: 09/01/2020, Positive, Last updated: 04/08/2021 -
List item
Orphan designation: autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene for: Treatment of sickle cell disease
Date of designation: 09/01/2020, Positive, Last updated: 25/01/2022 -
List item
Orphan designation: motixafortide for: Treatment of pancreatic cancer
Date of designation: 09/01/2020, Positive, Last updated: 19/05/2020 -
List item
Orphan designation: ziritaxestat for: Treatment of systemic sclerosis
Date of designation: 09/01/2020, Withdrawn, Last updated: 06/04/2021 -
List item
Orphan designation: Setmelanotide for: Treatment of Alström syndrome
Date of designation: 09/01/2020, Positive, Last updated: 01/07/2022 -
List item
Orphan designation: romilkimab for: Treatment of systemic sclerosis
Date of designation: 09/01/2020, Withdrawn, Last updated: 06/04/2021