Forschungsdatenbank

Suchergebnis Ansicht Alles (157) Projekte (39) Publikationen (117) 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 Choudhury Momoko, Lenders Malte, Laufer Pauline, Masthoff Max, Canaan-Kuehl Sima, Kurschat Christine, Muschol Nicole, Hennermann Julia B., Cybulla Markus, Kaufeld Jessica, Brand Eva, Bischof Antje Impact of migalastat on cerebral outcomes in fabry disease - results from the prospective observational FAMOUS trial NEUROLOGICAL RESEARCH AND PRACTICE. 2025; 7(1). Article Datensatz in Web of Science® Fu Chun-Yu, Kohl Joshua B., Liebsch Filip, D'Andrea Davide, Ditroi Tamas, Ogata Seiryo, Neuser Franziska, Mai Max, Mellis Anna T., Kouroussis Emilia, Morita Masanobu, Gehling Titus, Santamaria-Araujo Jose Angel, Yeo Sin yuin, Endepols Heike, Krizkova Michaela, Kozich Viktor, Krueger Marcus, Hennermann Julia B., Barayeu Uladzimir, Akaike Takaaki, Nagy Peter, Filipovic Milos, Schwarz Guenter Sulfite oxidase deficiency causes persulfidation loss and hydrogen sulfide release JOURNAL OF CLINICAL INVESTIGATION. 2025; 135(21). Article Datensatz in Web of Science® 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® Lee A, Bliksrud YT, Onali M, Neugebauer J, Eyskens F, Haas D, Moosler K, Enekwe A, Kiec-Wilk B, Diep LM, Bellettato CM, Zernikow B, Scarpa M, Rahman S, MetabERN collaboration group on palliative care, Hennermann JB, Tangeraas T Palliative Care for Children and Adults With Inherited Metabolic Disease in Europe: An Underutilised Service for Supportive Treatment and Care J Inherit Metab Dis. 2025; 48(e): 70095 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®

Vorträge Hennermann Julia B. Treatment of mucopolysaccharidoses Vortrag 2014