Seminar SeriesSelecting the right candidate: Which disease-causing variants are eligible for N=1 ASOs?
Articles & PodcastsQ&A: FDA's Peter Marks ready to encourage more accelerated approvals for rare diseases
Articles & Podcasts‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
Seminar SeriesFrom Mutation to Dose-able CRISPR Medicine In 3 Months: Obstacles & Actionable Paths Forward
