Forschungsdatenbank

Suchergebnis Ansicht Alles (151) Projekte (39) Publikationen (111) 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 Laufs V., Hasenburg A., Busch M. A., Lang F., Macchiella D., Mildenberger E., Seidmann L., Hennermann J. B. Placenta Pathologies in Two Patients With Glycogen Storage Disease Type Ia and Preeclampsia JIMD REPORTS. 2025; 66(4). Article Datensatz in Web of Science® Mengel Eugen, Da Riol Rosalia M., Del Toro Mireia, Deodato Federica, Gautschi Matthias, Grunewald Stephanie, Gronborg Sabine Weller, Harmatz Paul, Hennermann Julia B., Heron Benedicte, Maier Esther M., Santra Saikat, Sharma Reena, Tylki-Szymanska Anna, Cording Malene, Himmelstrup Louise, Guenther Sven, Dali ? Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial MOLECULAR GENETICS AND METABOLISM. 2025; 145(4). Article Datensatz in Web of Science® Mengel Eugen, Patterson Marc C., Da Riol Rosalia M., Del Toro Mireia, Deodato Federica, Gautschi Matthias, Grunewald Stephanie, Gronborg Sabine Weller, Harmatz Paul, Hennermann Julia B., Heronk Benedicte, Maier Esther M., Roubertie Agathe, Santra Saikat, Tylki-Szymanska Anna, LaGorio Lisa, Berry-Kravis Elizabeth, Porter Forbes D., Solomon Beth, Himmelstrup Louise, Mickle Travis, Guenther Sven, Dali Christine i Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale MOLECULAR GENETICS AND METABOLISM REPORTS. 2025; 43. Article Datensatz in Web of Science® Mistry Pramod K., Carwile Jenny L., Burrow Andrew, Ganesh Jaya, Camelo Jr Jose Simon, Hennermann Julia B., Batista Julie L., Oliveira-dos-Santos Antonio, Perichon Maria G., Tantawy Azza Long-term outcomes of imiglucerase treatment in children withGaucher disease type 1 or type 3 starting therapy before the age of2 years MOLECULAR GENETICS AND METABOLISM. 2025; 144(2). Meeting Abstract Datensatz in Web of Science® Muetze Ulrike, Stengel Julia, Gleich Florian, Kessler Svenja, Gruenert Sarah C., Thimm Eva, Hennermann Julia B., Freisinger Peter, Kraemer Johannes, Schnabel-Besson Elena, Mengler Katharina, Shtylla Alboren, Morath Marina, Hoffmann Georg F., Garbade Sven, Koelker Stefan Long-Term Outcomes of Adolescents and Young Adults Identified by Metabolic Newborn Screening PEDIATRICS. 2025; 155(4). Article Datensatz in Web of Science®

Vorträge Hennermann Julia B. Treatment of mucopolysaccharidoses Vortrag 2014