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Researchers propose a new approval blueprint for ultra-rare pediatric gene therapies
Key Takeaways
- Researchers argue that individualized pediatric gene therapies are advancing faster than traditional approval systems.
- The paper cites recent cases in which bespoke treatments were developed in about three years and eight months.
- Authors say the for-profit drug model is poorly suited to ultra-rare, patient-specific therapies.
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DT Editorial Team··via nature.com