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Muntau, Ania [Author]; Gramer, Gwendolyn [Author]

Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients <4 years

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  • Media type: E-Article
  • Title: Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients <4 years : results from the SPARK open-label, multicentre, randomized phase IIIb trial
  • Contributor: Muntau, Ania [Author]; Gramer, Gwendolyn [Author]
  • Published: 9 March 2017
  • Published in: Orphanet journal of rare diseases ; 12(2017), Artikel-ID 47, Seite 1-11
  • Language: English
  • DOI: 10.1186/s13023-017-0600-x
  • Identifier:
  • Origination:
  • Footnote:
  • Description: Sapropterin dihydrochloride, a synthetic formulation of BH4, the cofactor for phenylalanine hydroxylase (PAH, EC 1.14.16.1), was initially approved in Europe only for patients ≥4 years with BH4-responsive phenylketonuria. The aim of the SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) trial was to assess the efficacy (improvement in daily phenylalanine tolerance, neuromotor development and growth parameters), safety and pharmacokinetics of sapropterin dihydrochloride in children <4 years.
  • Access State: Open Access