Karl E. Anderson

TL;DR: The authors argue that the approach to designing and evaluating the success of translational training programs must be flexible enough to accommodate the needs of individual institutions and individual trainees within the institutions but that it must also be rigorous enough to document that the program is meeting its short-, intermediate-, and long-term objectives and that its trainees are meeting preestablished competency requirements.

TL;DR: The panel, which represents specialties including internal medicine, pediatrics, genetics, gastroenterology, hepatology, and hematology, was charged with formulating updated recommendations for diagnosing and treating the acute porphyrias, issued recommendations based on the specific enzyme and gene defects identified.

TL;DR: The disease is usually classified as sporadic if uroporphyrinogen decarboxylase is deficient only in the liver, and as familial (autosomal dominant) if the enzyme is also deficient in nonhepatic tissues such as erythrocytes.

TL;DR: Significant influences of nutritional factors on oxidative biotransformation of drugs in man are demonstrated, particularly when the subjects were placed on standard diets followed by the standard diets supplemented with carbohydrate or protein.

TL;DR: Givosiran led to lower levels of urinary ALA and porphobilinogen, fewer days of hemin use, and better daily scores for pain than placebo, and the increased efficacy was accompanied by a higher frequency of hepatic and renal adverse events.