Janus Kinase Inhibition in the Aicardi-Goutieres Syndrome

Adeline Vanderver,Laura Adang, Francesco Gavazzi, Katherine McDonald,Guy Helman,David B. Frank, Nicole Jaffe,Sabrina W. Yum,Abigail Collins, Stephanie R. Keller,Pierre Lebon, Jean-Francois Meritet, Jullie Rhee

NEW ENGLAND JOURNAL OF MEDICINE（2020）

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摘要

JAK Inhibition in the Aicardi–Goutieres Syndrome Patients with the Aicardi–Goutieres syndrome, an autosomal recessive disorder that affects the central nervous system, immune system, and skin, have...

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