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?

Oxford-Harrington Rare Disease Centre named first global initiative for children with rare disease

Pilot launched to support children with rare conditions to access personalised therapies

The world’s first pathway for individually designed drugs

Collaboratives, Companies Mobilize to BuildMomentum for 'N-of-1' ASO Therapies

Early Rare Disease Diagnosis Could Save as Much as $500,000 per Patient

What We Owe Terry Horgan: Reflections from Providers, Family, and Scientists

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.

‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine

The quest for the era of personalised medicine

A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments

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

Democratizing drug development will be a win for all