OTS Rare Disease N-of-1+ Briefing Document, Jan 2020
This briefing document aims to outline the current state of the art of oligonucleotide therapies for those planning to develop...
OTS Rare Disease N-of-1+ Briefing Document, Jan 2020
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Drug Regulation in the Era of Individualized Therapies
Drug tailored to one girl with brain disease paves way for similar customized treatments
This girl’s dramatic story shows hyper-personalized medicine is possible—and costly
After a bespoke therapy rescues a young girl, the FDA considers advance of individualized treatments
When ‘right to try’ isn’t enough: Congress wants a single ALS patient to get a therapy never tested