✦ LIBER ✦

Introduction of sapropterin dihydrochloride as standard of care in patients with phenylketonuria

✍ Scribed by H.J. Vernon; C.B. Koerner; M.R. Johnson; A. Bergner; A. Hamosh

Book ID: 116988990
Publisher: Elsevier Science
Year: 2010
Tongue: English
Weight: 231 KB
Volume: 100
Category: Article
ISSN: 1096-7192
DOI: 10.1016/j.ymgme.2010.03.022

No coin nor oath required. For personal study only.

📜 SIMILAR VOLUMES

Safety and efficacy of 22 weeks of treat

Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria

✍ Phillip Lee; Eileen P. Treacy; Eric Crombez; Melissa Wasserstein; Lewis Waber; J 📂 Article 📅 2008 🏛 John Wiley and Sons 🌐 English ⚖ 115 KB 👁 2 views

## Abstract Phenylketonuria (PKU) is an inherited metabolic disease characterized by phenylalanine (Phe) accumulation, which can lead to neurocognitive and neuromotor impairment. Sapropterin dihydrochloride, an FDA‐approved synthetic formulation of tetrahydrobiopterin (6R‐BH4, herein referred to as

Pharmacokinetics of Sapropterin in Patie

Pharmacokinetics of Sapropterin in Patients with Phenylketonuria

✍ Prof. François Feillet, Lorne Clarke, Concetta Meli, Mark Lipson, Andrew A. Morr 📂 Article 📅 2008 🏛 Springer International Publishing AG 🌐 English ⚖ 144 KB

Safety of extended treatment with saprop

Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: Results of a phase 3b study

✍ Barbara K. Burton; Maria Nowacka; Julia B. Hennermann; Mark Lipson; Dorothy K. G 📂 Article 📅 2011 🏛 Elsevier Science 🌐 English ⚖ 290 KB

START, a double blind, placebo-controlle

START, a double blind, placebo-controlled pharmacogenetic test of responsiveness to sapropterin dihydrochloride in phenylketonuria patients

✍ Jeanine R. Jarnes Utz; Cindy Pham Lorentz; Dorothy Markowitz; Kyle D. Rudser; Br 📂 Article 📅 2012 🏛 Elsevier Science 🌐 English ⚖ 270 KB

Sapropterin dihydrochloride use in pregn

Sapropterin dihydrochloride use in pregnant women with phenylketonuria: An interim report of the PKU MOMS sub-registry

✍ Grange, Dorothy K.; Hillman, Richard E.; Burton, Barbara K.; Yano, Shoji; Vockle 📂 Article 📅 2014 🏛 Elsevier Science 🌐 English ⚖ 219 KB

Sapropterin is associated with improveme

Sapropterin is associated with improvements in assessments of executive function in Canadian patients with phenylketonuria

✍ Deschênes, Elaine; Ryan, Lynn; Roy-Girard, Virginie 📂 Article 📅 2014 🏛 Elsevier Science 🌐 English ⚖ 60 KB