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Milasen
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease, Oct 2019
Drug Regulation in the Era of Individualized Therapies, Oct 2019
News Coverage:
Science
STAT
MIT Tech Review
Oligonucleotide Therapeutics Society
OTS Rare Disease N-of-1+ Briefing Document, Jan 2020
Afinersen
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide, Dec 2021
Nature Medicine
Jacifusen
Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis, Jan 2022
Atipeksen
Valeriasen
Gene Treatment for Rare Epilepsy Causes Brain Side Effect in 2 Children
