Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study
| Autor: | Trefz, Friedrich K. a, ⁎, Burton, Barbara K. b, Longo, Nicola c, Casanova, Mercedes Martinez-Pardo d, Gruskin, Daniel J. e, Dorenbaum, Alex f, Kakkis, Emil D. f, Crombez, Eric A. g, Grange, Dorothy K. h, Harmatz, Paul i, Lipson, Mark H. j, Milanowski, Andrzej k, Randolph, Linda Marie l, Vockley, Jerry m, Whitley, Chester B. n, Wolff, Jon A. o, Bebchuk, Judith p, Christ-Schmidt, Heidi p, Hennermann, Julia B. q |
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| Zdroj: | In The Journal of Pediatrics 2009 154(5):700-707 |
| Databáze: | ScienceDirect |
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