Metachromatic Leukodystrophy — Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Citation(s)
A Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human ARSA cDNA to Children With Metachromatic Leukodystrophy.