Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study.
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J. Vockley | B. Burton | N. Longo | L. Randolph | M. Casanova | D. Grange | C. Whitley | P. Harmatz | F. Trefz | J. Hennermann | M. Lipson | A. Dorenbaum | E. Crombez | H. Christ-schmidt | E. Kakkis | A. Milanowski | J. Wolff | J. Bebchuk | D. Gruskin