Perspectives on Clinical Outcomes for N=1 Patients: Insights from N1C Clinical Outcomes Workgroup

What if coding personalized ASO therapies only required the press of a button?

Tailored RNA therapies for ultrarare CNS diseases & mutations: a criteria framework for patient ID

Allele-specific ASO treatment for SCN2A-encephalopathy

Approach to Drafting Regulatory Submissions and Challenges of Institutional Implementation

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