Use of pegvaliase in the management of phenylketonuria: Case series of early experience in US clinics.

Autor: Adams D; Atlantic Health Morristown Medical Center, Morristown, NJ, USA., Andersson HC; Hayward Genetics Center, Tulane University Medical School, New Orleans, LA, USA., Bausell H; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA., Crivelly K; Hayward Genetics Center, Tulane University Medical School, New Orleans, LA, USA., Eggerding C; Cooper University Health Care, Camden, NJ, USA., Lah M; Indiana University School of Medicine, Indianapolis, IN, USA., Lilienstein J; BioMarin Pharmaceutical Inc., Novato, CA, USA., Lindstrom K; Phoenix Children's Hospital, Phoenix, AZ, USA., McNutt M; University of Texas Southwestern Medical Center, Dallas, TX, USA., Ray JW; University of Texas Medical Branch, Galveston, TX, USA., Saavedra H; The University of Texas Health Science Center at Houston - McGovern Medical School, Houston, TX, USA., Sacharow S; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Starin D; Rare Disease Institute, Children's National, Washington, DC, USA., Tiffany-Amaro J; St. Christopher's Hospital for Children, Philadelphia, PA, USA., Thomas J; University of Colorado School of Medicine, Aurora, CO, USA., Vucko E; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA., Wessenberg LB; OHSU Doernbecher Children's Hospital, Portland, OR, USA., Whitehall K; BioMarin Pharmaceutical Inc., Novato, CA, USA.

Publikováno v: Molecular genetics and metabolism reports [Mol Genet Metab Rep] 2021 Aug 14; Vol. 28, pp. 100790. Date of Electronic Publication: 2021 Aug 14 (Print Publication: 2021).