期刊
MOLECULAR GENETICS AND METABOLISM REPORTS
卷 24, 期 -, 页码 -出版社
ELSEVIER
DOI: 10.1016/j.ymgmr.2020.100603
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We present Boston Children's Hospital's clinic model for pegvaliase therapy in adults with phenylketonuria (PKU) and clinical outcomes in 46 patients over the first 1.5 years of commercial therapy. Approximately 70% (18/26) of patients starting pegvaliase achieved blood phenylalanine (Phe) < 360 mu mol/L, with an average of a 68 +/- 24% decrease in blood Phe from baseline. All patients experienced at least minor side effects, but in most, management of the side effects allowed for treatment to continue.
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