A Targeted Agent for Sickle Cell Disease - Changing the Protein but Not the Gene
- PMID: 31199089
- DOI: 10.1056/NEJMe1906771
A Targeted Agent for Sickle Cell Disease - Changing the Protein but Not the Gene
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A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14. N Engl J Med. 2019. PMID: 31199090 Clinical Trial.
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