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Community register of orphan medicinal products


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Vector based on an adeno-associated virus serotype 2 backbone, pseudo-serotyped with a type 8 capsid, which carries the coding sequence of the human TYMP gene under the control of the human thyroxine binding globulin promoter

EU orphan designation number: EU/3/14/1326   
Active ingredient: Vector based on an adeno-associated virus serotype 2 backbone, pseudo-serotyped with a type 8 capsid, which carries the coding sequence of the human TYMP gene under the control of the human thyroxine binding globulin promoter
Indication: Treatment of mitochondrial neurogastrointestinal encephalomyopathy
Sponsor: Vall d'Hebron Institute of Research
Pg. Vall d'Hebron 119-129, 08035 Barcelona, España

   Public summary of scientific opinion    

European Commission proceduresGoto top of the page
Close date procedure Procedure type EMEA number Decision summary publ decision docs annex
26/08/2014 Orphan designation EMA/OD/093/14 (2014)6131 of 22/08/2014