5-year retrospective analysis of patients with phenylketonuria (PKU) and hyperphenylalaninemia treated at two specialized clinics

Molecular Genetics and Metabolism - Tập 129 - Trang 177-185 - 2020
Harvey Levy1,2, Diana Lamppu3, Vera Anastosoaie1, Jennifer L. Baker4, Kevin DiBona5, Sarah Hawthorne1, Jessica Lindenberger4, Deborah Kinch3, Albert Seymour3, Mark McIlduff6, Sharon Watling6, Jerry Vockley4
1Division of Genetics and Genomics, Boston Children’s Hospital, Boston, MA, USA;
2Harvard Medical School, Boston, MA, USA
3Homology Medicines, Inc., Bedford, MA, USA
4Division Medical Genetics, Department of Pediatrics, University of Pittsburgh School of Medicine, UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, USA
5Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA
6Boston Biomedical Associates, LLC, Marlborough, MA, USA

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