Rare-disease researchers pioneer a unique approach to clinical trials

Personalized medicine is having its day

N-of-1 therapies: How clinical trials for a single patient can change the future

Individualized ASO therapy for rare diseases

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs

Democratizing drug development will be a win for all

They Created a Drug for Susannah. What About Millions of Other Patients?

Hopes for Antisense Oligonucleotides Remain Strong Despite Severe Adverse Events in Trials

We Can Cure Disease by Editing a Person’s DNA. Why Aren’t We?

Gene Treatment for Rare Epilepsy Causes Brain Side Effect in 2 Children

From Mila to millions

A mother, shaped by tragedy, embarks on a mission to advance custom medicines

New antisense oligonucleotide therapies reach first base in ALS

If DNA is like software, can we just fix the code?

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