Pediatric SDLT CNS Disorders research hub

Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions

This study looks into new, specially made medicines called ASOs for children aged 1 and up with very serious and life-threatening brain and nerve conditions caused by unique gene differences. The aim is to see if these medicines can help by correcting the gene problem.