دورية أكاديمية

Long-term efficacy and safety of sapropterin in patients who initiated sapropterin at < 4 years of age with phenylketonuria: results of the 3-year extension of the SPARK open-label, multicentre, randomised phase IIIb trial

التفاصيل البيبلوغرافية
العنوان: Long-term efficacy and safety of sapropterin in patients who initiated sapropterin at < 4 years of age with phenylketonuria: results of the 3-year extension of the SPARK open-label, multicentre, randomised phase IIIb trial
المؤلفون: Muntau, Ania C.Aff1, Burlina, Alberto, Eyskens, François, Freisinger, Peter, Leuzzi, Vincenzo, Sivri, Hatice Serap, Gramer, Gwendolyn, Pazdírková, Renata, Cleary, Maureen, Lotz-Havla, Amelia S., Lane, Paul, Alvarez, Ignacio, Rutsch, Frank
المصدر: Orphanet Journal of Rare Diseases. 16(1)
قاعدة البيانات: Springer Nature Journals
الوصف
تدمد:17501172
DOI:10.1186/s13023-021-01968-1