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

Data Sharing & an Open Letter

N of Small Trial with LBSL

The Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development

Models for Sustainable Discovery

An Overview of the N=1 Collaborative and ASO Treatment for KCNT1

Individualized ASO therapy for rare diseases

Hanging Our Hat on Shared Learnings