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SILEx December 2023: N=1 Ultra-Rare Projects: From Family Concern to Biotech. What Did We Learn?
Lauren Black will present on N=1 Ultra-Rare Projects.
- Family situations that drive them to seeking individualized or genetic therapies
- Challenges this poses to “normal” drug development – need speed, quality and limited cost
- Some examples of how many divisions in CRL have helped advance cutting edge medicines
Intended audience Veterinary staff members and Study Directors.