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

U.S., Feb. 14 -- ClinicalTrials.gov registry received information related to the study (NCT07410143) titled 'Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions' on Jan. 12.

Brief Summary: This study is being conducted to evaluate individualised antisense oligonucleotides (ASOs) in participants with severely debilitating, life threatening (SDLT) central nervous system (CNS) conditions caused by unique genetic variants amenable to correction by an ASO.

Study Start Date: Jan. 13

Study Type: INTERVENTIONAL

Condition: Pediatric CNS

Intervention: DRUG: Individualized ASO

Individualized ASO

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