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Accelerated approval will be 'the norm' for gene therapies, FDA's Peter Marks says
Four Keys To Advancing Antisense Oligonucleotides For Personalized Medicine
This lifesaving treatment was designed for one. Could it be the future of medical care?
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
