GM1 Gangliosidosis

  • P1-GM-101 : An open-label adaptative design study of intracisternal administration of Aden-associated viral vector serotype rh.10 carrying the human β-galactosidase cDNA for the treatment of GM1 gangliosidosis




Intervention LYS-GM101
Principal investigator Bénédicte Héron
Sponsor Lysogene
Study status In preparation
Recruitment status In preparation
Population Child
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