A study testing agene therapy to treat mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) A study testing agenetherapy to treat mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrom ...
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: CRYOPRESERVEDAUTOLOGOUSCD34+CELLSTRANSDUCEDWITHALENTIVIRALVECTORCONTAININGHUMANSGSHGENE INN or Proposed INN: no recommended INN Other descriptive name: AUTOLOGOUSCD34+CELLSTRANSDUCEDWITHALENTIVIRALVECTORCONTAINING THE HUMANSGSHGENEProduct Name: CRYOPRESERVEDAUTOLOGOUSCD34+CELLSTRANSDUCEDWITHALENTIVIRALVECTORCONTAININGHU ...