✦ LIBER ✦

Use of sapropterin dihydrochloride in maternal phenylketonuria. A European experience of eight cases

✍ Scribed by Feillet, François; Muntau, Ania C.; Debray, François-Guillaume; Lotz-Havla, Amelie S.; Puchwein-Schwepcke, Alexandra; Fofou-Caillierez, Ma’atem Béatrice; van Spronsen, Francjan; Trefz, Fritz Friedrich

Book ID: 125356709
Publisher: Springer
Year: 2014
Tongue: English
Weight: 590 KB
Volume: 37
Category: Article
ISSN: 0141-8955
DOI: 10.1007/s10545-014-9716-5

No coin nor oath required. For personal study only.

📜 SIMILAR VOLUMES

Longitudinal quality of life analysis in

Longitudinal quality of life analysis in a phenylketonuria cohort provided sapropterin dihydrochloride

✍ Douglas, Teresa D; Ramakrishnan, Usha; Kable, Julie A; Singh, Rani H 📂 Article 📅 2013 🏛 BioMed Central 🌐 English ⚖ 427 KB

Introduction of sapropterin dihydrochlor

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

✍ H.J. Vernon; C.B. Koerner; M.R. Johnson; A. Bergner; A. Hamosh 📂 Article 📅 2010 🏛 Elsevier Science 🌐 English ⚖ 231 KB

Use of sapropterin in the management of

Use of sapropterin in the management of phenylketonuria: Seven case reports

✍ Gokmen Ozel, H.; Lammardo, A.M.; Motzfeldt, K.; Robert, M.; Rocha, J.C.; van Rij 📂 Article 📅 2013 🏛 Elsevier Science 🌐 English ⚖ 135 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

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

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