Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
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. A Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication ...
Metachromatic Leukodystrophy
Genetic: intracerebral administration of AAVrh.10cuARSA
Institut National de la Santé Et de la Recherche Médicale, France
European Leukodystrophy Association;Assistance Publique - Hôpitaux de Paris
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 - Intracerebral Gene Therapy for MLD A phase I/II, open labeled, monocentric study of direct intracranial administration of a replication ...
Early onset forms of MLD MedDRA version: 14.1;Level: SOC;Classification code 10029205;Term: Nervous system disorders;System Organ Class: 10029205 - Nervous system disorders MedDRA version: 14.1;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Early onset forms of MLD MedDRA version: 14.1;Level: SOC;Classification code 10029205;Term: Nervous ...
Product Name: AAVrh.10cuARSA Product Code: Non applicable