Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
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N Engl J Med. 2019 Oct 24;381(17):1644-1652. doi: 10.1056/NEJMoa1813279. Epub 2019 Oct 9.
N Engl J Med. 2019.
PMID: 31597037
Free PMC article.