- 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
Acronym |
P1-GM-101 |
| Intervention | LYS-GM101 |
| Principal investigator | Bénédicte Héron |
| Sponsor | Lysogene |
| Study status | Active |
| Recruitment status | Ongoing |
| Population | Child |
| + infos on clinicaltrials.gov |
