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Results showing a 74% reduction in seizures set up Stoke for a pivotal study and could make STK-001 the first disease-modifying agent for the condition.

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Bespoke Gene Therapy ‘Playbook’ Outlines Platform Approach To AAV-Based Treatments

Supporting ‘n of 1’ gene therapy development, the playbook from a Foundation for the National Institutes of Health-backed consortium aims to help investigators who are not traditional product sponsors prepare for meetings with US FDA and IND submissions.

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Stark Warnings For Pharma On Preserving Trust And Overcoming Risk Aversion

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