Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy

NCT01801709 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

5

Enrollment

OTHER_GOV

Sponsor class

Conditions

Metachromatic Leukodystrophy

Interventions

GENETIC: intracerebral administration of AAVrh.10cuARSA

Sponsor

Institut National de la Santé Et de la Recherche Médicale, France

Collaborators

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