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?

Rare-disease researchers pioneer a unique approach to clinical trials

Sharing Papers, Presentations and More

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

Personalized medicine is having its day