Publications by authors named "Zheng Jason Yuan"

Article Synopsis
  • The study investigates the clinical efficacy of the drug combination lumacaftor/ivacaftor (LUM/IVA) for patients with cystic fibrosis (CF) who have a rare CFTR mutation (A455E-CFTR).
  • Twenty participants were randomized to receive either LUM/IVA or a placebo, with the primary goal of measuring changes in lung function and sweat chloride levels over eight weeks.
  • While LUM/IVA did not show a significant improvement in lung function (ppFEV), it did result in a notable decrease in sweat chloride concentration, indicating some efficacy in treating patients with the A455E-CFTR mutation.
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Pyruvate kinase deficiency is a chronic hemolytic anemia caused by mutations in PK-R, a key glycolytic enzyme in erythrocytes. These 2 phase 1 randomized, placebo-controlled, double-blind healthy-volunteer studies assessed the safety, tolerability, and pharmacokinetics/pharmacodynamics of AG-348, a first-in-class allosteric PK-R activator. Twelve sequential cohorts were randomized 2:6 to receive oral placebo or AG-348, respectively, as a single dose (30-2500 mg) in the single-ascending-dose (SAD) study (ClinicalTrials.

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