| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10833771 | Molecular Genetics and Metabolism | 2012 | 8 Pages |
Abstract
⺠Metabolic experts convened to draft recommendations for sapropterin use in PKU. ⺠Our aim is to provide a uniform and practical approach for sapropterin use in PKU. ⺠Recommendations are based on extensive clinical experience and current evidence. ⺠Guidance from patient selection to long-term management of patients is presented. ⺠This could serve as a useful adjunct to future national consensus statements.
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Authors
Amy Cunningham, Heather Bausell, Mary Brown, Maggie Chapman, Kari DeFouw, Sharon Ernst, Julie McClure, Helen McCune, Donna O'Steen, Amy Pender, Jill Skrabal, Ann Wessel, Elaina Jurecki, Renée Shediac, Suyash Prasad, Jane Gillis, Stephen Cederbaum,