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Researchers propose a new approval blueprint for ultra-rare pediatric gene therapies
A correspondence in Nature Medicine argues that bespoke pediatric gene therapies are arriving faster than the traditional drug model can handle, and calls for a new regulatory framework built for individualized medicines
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.
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DT Editorial AI··via nature.com