Sapropterin dihydrochloride for phenylketonuria

2010 ◽  
Author(s):  
Usha Rani Somaraju ◽  
Marcus Merrin
Keyword(s):  
Sapropterin Dihydrochloride

Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study

2009 ◽  
Vol 154 (5) ◽  
pp. 700-707.e1 ◽  
Author(s):  
Friedrich K. Trefz ◽  
Barbara K. Burton ◽  
Nicola Longo ◽  
Mercedes Martinez-Pardo Casanova ◽  
Daniel J. Gruskin ◽  
...  
Keyword(s):  
Phase Iii ◽  
Controlled Study ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Sapropterin Dihydrochloride

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

2011 ◽  
Vol 103 (4) ◽  
pp. 315-322 ◽  
Author(s):  
Barbara K. Burton ◽  
Maria Nowacka ◽  
Julia B. Hennermann ◽  
Mark Lipson ◽  
Dorothy K. Grange ◽  
...  
Keyword(s):  
Extended Treatment ◽  
Sapropterin Dihydrochloride

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

2014 ◽  
Vol 37 (5) ◽  
pp. 753-762 ◽  
Author(s):  
François Feillet ◽  
Ania C. Muntau ◽  
François-Guillaume Debray ◽  
Amelie S. Lotz-Havla ◽  
Alexandra Puchwein-Schwepcke ◽  
...  
Keyword(s):  
Maternal Phenylketonuria ◽  
Sapropterin Dihydrochloride ◽  
European Experience

scholarly journals International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria

2019 ◽  
Vol 127 (1) ◽  
pp. 1-11 ◽  
Author(s):  
Ania C. Muntau ◽  
Darius J. Adams ◽  
Amaya Bélanger-Quintana ◽  
Tatiana V. Bushueva ◽  
Roberto Cerone ◽  
...  
Keyword(s):  
Best Practice ◽  
Sapropterin Dihydrochloride
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