Gene therapy study with autologous hemapoietic stem cells for patients affected by MPSIH
A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human a-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant - TigetT10_MPS1H A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor ce ...
Mucopolysaccharidosis type I Hurler MedDRA version: 20.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] Mucopolysaccharidosis type I Hurler MedDRA version: 20.1;Level: PT;Classification code 10056886;Term ...