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Veröffentlichungsdatum
Anzahl der Publikationen: 3
2021
Muntau, Ania C.; Burlina, Alberto; Eyskens, Francois; Freisinger, Peter; Leuzzi, Vincenzo; Sivri, Hatice Serap; Gramer, Gwendolyn; Pazdirkova, Renata; Cleary, Maureen; Lotz-Havla, Amelia S.; Lane, Paul; Alvarez, Ignacio und Rutsch, Frank
(2021):
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.
In: Orphanet Journal of Rare Diseases, Bd. 16, Nr. 1, 341
2020
Pinto, Alex; Evans, Sharon; Daly, Anne; Almeida, Manuela Ferreira; Assoun, Murielle; Belanger-Quintana, Amaya; Bernabei, Silvia Maria; Bollhalder, Sandra; Cassiman, David; Champion, Helena; Chan, Heidi; Corthouts, Karen; Dalmau, Jaime; Boer, Foekje de; Laet, Corinne de; Meyer, An de; Desloovere, An; Dianin, Alice; Dixon, Marjorie; Dokoupil, Katharina; Dubois, Sandrine; Eyskens, Francois; Faria, Ana; Fasan, Ilaria; Favre, Elisabeth; Feillet, Francois; Fekete, Anna; Gallo, Giorgia; Gingell, Cerys; Gribben, Joanna; Hansen, Kit Kaalund; Ter Horst, Nienke; Jankowski, Camille; Janssen-Regelink, Renske; Jones, Ilana; Jouault, Catherine; Kahrs, Gudrun Elise; Kok, Irene; Kowalik, Agnieszka; Laguerre, Catherine; Le Verge, Sandrine; Liguori, Alessandra; Lilje, Rina; Maddalon, Cornelia; Mayr, Doris; Meyer, Uta; Micciche, Avril; Och, Ulrike; Robert, Martine; Rocha, Julio Cesar; Rogozinski, Hazel; Rohde, Carmen; Ross, Kathleen; Saruggia, Isabelle; Schlune, Andrea; Singleton, Kath; Sjoqvist, Elisabeth; Skeath, Rachel; Stolen, Linn Helene; Terry, Allyson; Timmer, Corrie; Tomlinson, Lyndsey; Tooke, Alison; Vande Kerckhove, Kristel; Dam, Esther van; Hurk, Dorine van den; Ploeg, Liesbeth van der; Driessche, Marleen van; Rijn, Margreet van; Wegberg, Annemiek van; Vasconcelos, Carla; Vestergaard, Helle; Vitoria, Isidro; Webster, Diana; White, Fiona; White, Lucy; Zweers, Heidi und MacDonald, Anita
(2020):
Dietary practices in methylmalonic acidaemia: a European survey.
In: Journal of Pediatric Endocrinology & Metabolism, Bd. 33, Nr. 1: S. 147-155
2017
Muntau, Ania C.; Burlina, Alberto; Eyskens, Francois; Freisinger, Peter; Laet, Corinne de; Leuzzi, Vincenzo; Rutsch, Frank; Sivri, H. Serap; Vijay, Suresh; Bal, Milva Orquidea; Gramer, Gwendolyn; Pazdirkova, Renata; Cleary, Maureen; Lotz-Havla, Amelie S.; Munafo, Alain; Mould, Diane R.; Moreau-Stucker, Flavie und Rogoff, Daniela
(2017):
Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients < 4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial.
In: Orphanet Journal of Rare Diseases
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