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Lindegren ML, Krishnaswami S, Reimschisel T, Fonnesbeck C, Sathe NA, McPheeters ML. A Systematic Review of BH4 (Sapropterin) for the Adjuvant Treatment of Phenylketonuria. JIMD reports. 8(8). 109-19.
Abstract
Dietary management is the mainstay of effective treatment in PKU, but dietary restriction is difficult and additional treatment options are needed.