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. 2022 Sep-Oct;137(1-2):107-113.
doi: 10.1016/j.ymgme.2022.07.015. Epub 2022 Aug 2.

The transformation of drug development for the 21st century: Time for a change

Emil D Kakkis  1
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Free article

The transformation of drug development for the 21st century: Time for a change

Emil D Kakkis. Mol Genet Metab. 2022 Sep-Oct.
Free article
No abstract available

Keywords: Accelerated approval; Biomarker; Drug development; Primary disease activity biomarker; Rare disease.

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