Antisense Oligonucleotide Clinical Guidance Emphasizes Individualized Nature Of Treatment

 
• By Sue Sutter

US FDA’ s latest ‘n of 1’ guidance describes clinical considerations for INDs to support dosing and monitoring of an individual with a life-threatening genetic disease who is eligible to receive an ASO; recommendations apply only when a few patients may be candidates for treatment.

Flowers in field
In FDA's view, a study for an individualized ASO therapy will involve 1 or 2 patients. • Source: Alamy

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