4 Einträge gefunden

1. A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A

   1. Wijburg, Frits A
   2. Whitley, Chester B
   3. Muenzer, Joseph
   4. Gasperini, Serena
   5. Del Toro, Mireia
   6. Muschol, Nicole
   7. Cleary, Maureen

   2021 - Forschungsinformationssystem des UKE - frei zugänglich
2. 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

   1. Muntau, Ania C
   2. Burlina, Alberto
   3. Eyskens, François
   4. Freisinger, Peter
   5. Leuzzi, Vincenzo
   6. Sivri, Hatice Serap
   7. Gramer, Gwendolyn

   2021 - Forschungsinformationssystem des UKE - frei zugänglich
3. Diagnostic and management practices for phenylketonuria in 19 countries of the South and Eastern European Region: survey results: survey results

   1. Giżewska, Maria
   2. MacDonald, Anita
   3. Bélanger-Quintana, Amaya
   4. Burlina, Alberto
   5. Cleary, Maureen
   6. Coşkun, Turgay
   7. Feillet, François

   2016 - Forschungsinformationssystem des UKE
4. Fluctuations in phenylalanine concentrations in phenylketonuria: a review of possible relationships with outcomes

   1. Cleary, Maureen
   2. Trefz, Friedrich
   3. Muntau, Ania C
   4. Feillet, François
   5. van Spronsen, Francjan J
   6. Burlina, Alberto
   7. Bélanger-Quintana, Amaya

   2013 - Forschungsinformationssystem des UKE