Articles & PodcastsA young girl’s custom gene therapy hints at a framework for tailored rare disease treatments
Seminar SeriesPerspectives on Clinical Outcomes for N=1 Patients: Insights from N1C Clinical Outcomes Workgroup
Seminar SeriesTailored RNA therapies for ultrarare CNS diseases & mutations: a criteria framework for patient ID
Seminar SeriesApproach to Drafting Regulatory Submissions and Challenges of Institutional Implementation
