GeneTherapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA GeneTherapy WithModified AutologousHematopoietic Stem Cellsfor Patients WithMucopolysaccharidosis T ...
A Phase I-II, Study of AutologousCD34+ Haematopoietic Stem CellsTransduced ex Vivo With CD11b LentiviralVector Encoding for HumanSGSH in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIa, Sanfilippo Syndrome Type A) A Phase I-II, Study of AutologousCD34+Haematopoietic Stem CellsTransducedex Vivo WithCD11b Lentivi ...
Orchard Therapeutics;CTI Clinical Trial and Consulting Services;University College, London;Great Ormond Street Hospital for Children NHS Foundation Trust;Manchester University NHS Foundation Trust Orchard Therapeutics;CTI Clinical Trial and Consulting Services;University College, London;Great Orm ...
A study testing agenetherapy to treat mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) A study testing agenetherapy to treat mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndro ...
A phase I-II, study of autologousCD34+ haematopoietic stem cellstransduced ex vivo with CD11b lentiviralvector encoding for humanSGSH in patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) A phase I-II, study of autologousCD34+haematopoietic stem cellstransducedex vivo withCD11b lentivi ...
MPS IIIA, mucopolysaccharidosis type IIIA MedDRA version: 20.1;Level: PT;Classification code 10028093;Term: Mucopolysaccharidosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] MPS IIIA, mucopolysaccharidosis type IIIA MedDRA version: 20.1;Level: PT;Classification code 1002809 ...
Product Name: CRYOPRESERVED AUTOLOGOUSCD34+CELLSTRANSDUCEDWITHALENTIVIRALVECTORCONTAININGHUMANSGSHGENE INN or Proposed INN: no recommended INN Other descriptive name: AUTOLOGOUSCD34+CELLSTRANSDUCEDWITHALENTIVIRALVECTORCONTAININGTHEHUMANSGSHGENEProduct Name: CRYOPRESERVED AUTOLOGOUSCD34+CELLSTRANSDUCEDWITHALENTIVIRALVECTORCONTAININGHU ...