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MPS IIIA phase I

Long-term Follow-up of MPS IIIA Patients Treated by Intercerebral LYS SAF301 Gene Therapy
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Now recruiting

MPS IIIA Observational

Long-term Follow-up of MPS IIIA Patients Treated by Intercerebral LYS SAF301 Gene Therapy
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Not yet recruiting

MPS IIIA Pivotal phase II/III

Long-term Follow-up of MPS IIIA Patients Treated by Intercerebral LYS SAF301 Gene Therapy
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GM1 Phase I

Phase I/II gene transfer clinical trial for GM1 gangliosidosis delivering LYS-GM101
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