AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human CLN2 cDNA to Children With LINCL With Uncommon Genotypes and/or Moderate to Severe Impairment Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Ser ...
Late Infantile Neuronal Ceroid Lipofuscinosis;Batten Disease