Our Story
Our Work
Who We Are
News + Seminars
Collaborating Organizations
Data Sharing
More
Articles & Media
Individualised medicines offer hope for children with a rare disease
Q&A: FDA's Peter Marks ready to encourage more accelerated approvals for rare diseases
Too many treatable diseases go unnoticed. This could change that.
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
