Journal of Pediatric Neurology and Medicine

Neurologic Paediatric Disease and Gene Therapy

Abstract

Renée Shellhaas*

For newborns and kids with nervous system disorders, gene-targeted medicines are now a reality. Rapid scientific progress has produced therapies that can cure or even change illness. Unresolved issues, however, include the necessity for long-term surveillance, inequities in delivery and delays and gaps in diagnosis.