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This lifesaving treatment was designed for one. Could it be the future of medical care?

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Oxford-Harrington Rare Disease Centre named first global initiative for children with rare disease

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Pilot launched to support children with rare conditions to access personalised therapies

Genomics England November 22, 2023 New programme announced to develop a pathway for children with rare conditions to access therapies...

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The world’s first pathway for individually designed drugs

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Community Events

Updated November 28, 2023 FDA Clinical Investigator Training Course (CITC) 2023: December 6-7 2023 ABOUT THIS EVENT This course is...

A Multistakeholder Perspective on Advancing Individualized Therapeutics

Articles & Podcasts

Collaboratives, Companies Mobilize to BuildMomentum for 'N-of-1' ASO Therapies

Regulatory: The Future Pathway to Access

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