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Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naive and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose studyNEUROMUSCULAR DISORDERS. Bd. 29. H. 3. 2019 S. 167-186
Ernst, Mareike; Tibubos, Ana N.; Unterrainer, Josef et al.
Status and predictors of planning ability in adult long-term survivors of CNS tumors and other types of childhood cancerSCIENTIFIC REPORTS. Bd. 9. 2019
Azabdaftari, Aline; van der Giet, Markus; Schuchardt, Mirjam et al.
The cardiovascular phenotype of adult patients with phenylketonuriaORPHANET JOURNAL OF RARE DISEASES. Bd. 14. H. 1. 2019
Lignitz, Sarah; Coors, Detlef; Pohlenz, Joachim
The challenge to treat neonatal autoimmune hyperthyroidism in a small pretermHORMONE RESEARCH IN PAEDIATRICS. Bd. 91. 2019 S. 614-614
Arash-Kaps, Laila; Komlosi, Katalin; Seegraeber, Marlene et al.
The Clinical and Molecular Spectrum of GM1 GangliosidosisJOURNAL OF PEDIATRICS. Bd. 215. 2019 S. 152-+
Ernst, Mareike; Braehler, Elmar; Wild, Philipp S. et al.
The desire for children among adult survivors of childhood cancer: Psychometric evaluation of a cancer-specific questionnaire and relations with sociodemographic and psychological characteristicsPSYCHO-ONCOLOGY. 2019
Guffon, Nathalie; Muschol, Nicole M.; Konstantopoulou, Vassiliki et al.
The first study investigating safety and efficacy of velmanase alfa (human recombinant alpha mannosidase) in alpha-mannosidosis patients below six years of ageMOLECULAR GENETICS AND METABOLISM. Bd. 126. H. 2. 2019 S. S65-S65
Hennermann, Julia B.; Guffon, Nathalie; Cattaneo, Federica et al.
The SPARKLE study: Shedding light on alpha mannosidosisMOLECULAR GENETICS AND METABOLISM. Bd. 126. H. 2. 2019 S. S71-S72
Kowalzik, Frank; Zepp, Fred
Update: STIKO vaccination recommendations for children and adolescentsMONATSSCHRIFT KINDERHEILKUNDE. Bd. 167. H. 3. 2019 S. 183-195
Wagner, Norbert; Assmus, Frauke; Arendt, Gabriele et al.
Vaccinate Patients with Immunodeficiency Instructions for Use of Vaccines recommended by the Standing Vaccination Commission (IV) Vaccination for Patients with Autoimmune Diseases, other chronic inflammatory Diseases and under immunomodulatory TherapyBUNDESGESUNDHEITSBLATT-GESUNDHEITSFORSCHUNG-GESUNDHEITSSCHUTZ. Bd. 62. H. 4. 2019 S. 494-515