Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies

This study evaluates the proportion of established antisense oligonucleotides (ASO) assessment guidelines that are amenable to ASO therapy approaches for genetically diagnosed infants.

Corresponding Author: Alissa M. D’Gama, MD, PhD, Division of Newborn Medicine, Department of Pediatrics, Boston Children’s Hospital, 300 Longwood Ave, Enders 961, Boston, MA 02115 (alissa.dgama@childrens.harvard.edu).

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