4 Einträge gefunden

1. Investigating health-related quality of life in rare diseases:a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

   1. Gissen, Paul
   2. Specchio, Nicola
   3. Olaye, Andrew
   4. Jain, Mohit
   5. Butt, Thomas
   6. Ghosh, Wrik
   7. Ruban-Fell, Benjamin

   2021 - Forschungsinformationssystem des UKE - frei zugänglich
2. Cerliponase Alfa for the Treatment of Atypical Phenotypes of CLN2 Disease:A Retrospective Case Series

   1. Wibbeler, Eva
   2. Wang, Raymond
   3. Reyes, Emily de Los
   4. Specchio, Nicola
   5. Gissen, Paul
   6. Guelbert, Norberto
   7. Nickel, Miriam

   2021 - Forschungsinformationssystem des UKE - frei zugänglich
3. Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration

   1. Kim, Aryun
   2. Grover, Anita
   3. Hammon, Kevin
   4. de Hart, Greg
   5. Slasor, Peter
   6. Cherukuri, Anu
   7. Ajayi, Temitayo

   2021 - Forschungsinformationssystem des UKE - frei zugänglich
4. Study of Intraventricular Cerliponase Alfa for CLN2 Disease

   1. Schulz, Angela
   2. Ajayi, Temitayo
   3. Specchio, Nicola
   4. de Los Reyes, Emily
   5. Gissen, Paul
   6. Ballon, Douglas
   7. Dyke, Jonathan P

   2018 - Forschungsinformationssystem des UKE