Forschungsdatenbank

Suchergebnis Ansicht Alles (145) Projekte (39) Publikationen (105) 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 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® Muschol Nicole, Hennermann Julia B., Magner Martin, Lampe Christina, Lopez-Rodriguez Monica A., Jurickova Katarina, Garavaglia Daniela, Dona Francesca, Donati Chiara, Fichera Vito, Guffon Nathalie Clinical profiles of 134 patients with alpha-mannosidosis from the velmanase alfa clinical program and SPARKLE registry MOLECULAR GENETICS AND METABOLISM. 2025; 144(2). Meeting Abstract Datensatz in Web of Science® Wasserstein Melissa, Gallagher Renata C., Barbato Antonio, Guigliani Roberto, Guelbert Norberto, Hennermann Julia B., Ikezoe Takayuki, Lachmann Robin, Lidove Olivier, Mabe Paulina, Mengel Eugen, Scarpa Maurizio, Senates Ebubekir, Tchan Michel, Villarrubia Jesus, Thurberg Beth L., Armstrong Nicole M., Srivastav Shruti, Wisniewska Urszula, Kumar Monica Final results of the ASCEND trial: Continued or sustained improvements in hepatosplenomegaly, respiratory outcomes, and lipid profile after 4 years of olipudase alfa enzyme replacement therapy in adults with acid sphingomyelinase deficiency MOLECULAR GENETICS AND METABOLISM. 2025; 144(2). Meeting Abstract Datensatz in Web of Science® Giugliani Roberto, Gonzalez-Meneses Antonio, Scarpa Maurizio, Burton Barbara, Wang Raymond, Martins Esmeralda, Oussoren Esmeralda, Hennermann Julia B., Chabrol Brigitte, Grant Christina L., Sun Angela, Durand Consuelo, Hetzer Joel, Malkus Betsy, Marsden Deborah, Merritt II J. Lawrence Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program ORPHANET JOURNAL OF RARE DISEASES. 2024; 19(1). Article Datensatz in Web of Science® Guffon Nathalie, Burton Barbara K., Ficicioglu Can, Magner Martin, Gil-Campos Mercedes, Lopez-Rodriguez Monica A., Jayakar Parul, Lund Allan M., Tal Galit, Garcia-Ortiz Jose Elias, Stepien Karolina M., Ellaway Carolyn, Al-Hertani Walla, Giugliani Roberto, Cathey Sara S., Hennermann Julia B., Lampe Christina, McNutt Markey, Lagler Florian B., Scarpa Maurizio, Sutton V. Reid, Muschol Nicole Monitoring and integrated care coordination of patients with alpha-mannosidosis: A global Delphi consensus study MOLECULAR GENETICS AND METABOLISM. 2024; 142(4). Article Datensatz in Web of Science®

Vorträge Hennermann Julia B. Treatment of mucopolysaccharidoses Vortrag 2014