Forschungsdatenbank

Suchergebnis Ansicht Alles (129) Projekte (39) Publikationen (89) Vorträge (1)

Projekte Sortiert Jahr Name Thema Hennermann Julia A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) with Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design Projekt 2022-2028 Hennermann Julia A PHASE III STUDY OF JR-141 IN MUCOPOLYSACCHARIDOSIS TYPE II (HUNTER SYNDROME) PATIENTS Projekt 2022-2025 Hennermann Julia SHP675-301: A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease Projekt 2021-2025 Hennermann Julia A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled 3-Part Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease Projekt 2018-2025 Hennermann Julia A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease Projekt 2018-2025

Publikationen Sortiert Jahr Name Titel Batsu Isabela, Zheng Riliang, Minini Pascal, Cherkasov Dmitry, Scott David, Goker-Alpan Ozlem, Hennermann Julia B., Sakurai Ken, Cox Timothy M. Two years of venglustat combined with imiglucerase shows continued positive effects on neurological features and brain connectivity in adults with Gaucher disease type 3 MOLECULAR GENETICS AND METABOLISM. 2023; 138(2): 15-15. Meeting Abstract Datensatz in Web of Science® Guffon Nathalie, Konstantopoulou Vassiliki, Hennermann Julia B., Muschol Nicole, Bruno Irene, Tummolo Albina, Ceravolo Ferdinando, Zardi Giulia, Ballabeni Andrea, Lund Allan Long-term safety and efficacy of velmanase alfa treatment in children under 6 years of age with alpha-mannosidosis: A phase 2, open label, multicenter study JOURNAL OF INHERITED METABOLIC DISEASE. 2023; 46(4): 705-719. Article Datensatz in Web of Science® Guffon Nathalie, Muschol Nicole M., Ballabeni Andrea, Borgwardt Line, Lund Allan, Gil-Campos Mercedes, Dona Francesca, Hennermann Julia B. Baseline characteristics of a real-world population with alpha-mannosidosis: Insights from the SPARKLE registry MOLECULAR GENETICS AND METABOLISM. 2023; 138(2): 54-54. Meeting Abstract Datensatz in Web of Science® Maier Esther M., Muetze Ulrike, Janzen Nils, Steuerwald Ulrike, Nennstiel Uta, Odenwald Birgit, Schuhmann Elfriede, Lotz-Havla Amelie S., Weiss Katharina J., Hammersen Johanna, Weigel Corina, Thimm Eva, Gruenert Sarah C., Hennermann Julia B., Freisinger Peter, Kraemer Johannes, Das Anibh M., Illsinger Sabine, Gramer Gwendolyn, Fang-Hoffmann Junmin, Garbade Sven F., Okun Juergen G., Hoffmann Georg F., Koelker Stefan, Roeschinger Wulf Collaborative evaluation study on 18 candidate diseases for newborn screening in 1.77 million samples JOURNAL OF INHERITED METABOLIC DISEASE. 2023. Article Datensatz in Web of Science® Muetze Ulrike, Henze Lucy, Schroeter Julian, Gleich Florian, Lindner Martin, Gruenert Sarah C., Spiekerkoetter Ute, Santer Rene, Thimm Eva, Ensenauer Regina, Weigel Johannes, Beblo Skadi, Arelin Maria, Hennermann Julia B., Marquardt Iris, Freisinger Peter, Kraemer Johannes, Dieckmann Andrea, Weinhold Natalie, Schiergens Katharina A., Maier Esther M., Hoffmann Georg F., Garbade Sven F., Koelker Stefan Isovaleric aciduria identified by newborn screening: Strategies to predict disease severity and stratify treatment JOURNAL OF INHERITED METABOLIC DISEASE. 2023. Article Datensatz in Web of Science®

Vorträge Hennermann Julia B. Treatment of mucopolysaccharidoses Vortrag 2014