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- Knuf, Markus
- Meningococcal vaccination What is new?
- MONATSSCHRIFT KINDERHEILKUNDE. Bd. 172. H. 5. 2024 S. 386-395
- Muschol, Nicole M.; Burton, Barbara K.; Ficicioglu, Can et al.
- A Delphi consensus approach to monitoring and integrated care coordination of patients with alpha-mannosidosis
- MOLECULAR GENETICS AND METABOLISM. Bd. 141. H. 2. 2024
- Stepien, Karolina M.; Hennermann, Julia B.; Curiati, Marco A. et al.
- Case series on patients with delayed diagnosis of mild/moderate alpha-mannosidosis
- MOLECULAR GENETICS AND METABOLISM. Bd. 141. H. 2. 2024
- Giugliani, Roberto; Gonzalez-Meneses, Antonio; Scarpa, Maurizio et al.
- 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. Bd. 19. H. 1. 2024
- Truninger, Moritz Ilan; Werner, Helene; Landolt, Markus Andreas et al.
- Living with Pompe disease: results from a qualitative interview study with children and adolescents and their caregivers
- ORPHANET JOURNAL OF RARE DISEASES. Bd. 19. H. 1. 2024
- Pfrimmer, Charlotte; Smitka, Martin; Muschol, Nicole et al.
- Long-Term Outcome of Infantile Onset Pompe Disease Patients Treated with Enzyme Replacement Therapy - Data from a German-Austrian Cohort
- JOURNAL OF NEUROMUSCULAR DISEASES. Bd. 11. H. 1. 2024 S. 167-177
- Schmitz, Lucia Segura; Hennermann, Julia B.; Lollert, Andre
- Lysosomal storage disorders - Fabry disease and Gaucher disease
- DEUTSCHE MEDIZINISCHE WOCHENSCHRIFT. Bd. 149. H. 21. 2024 S. 1263-1269
- Guffon, Nathalie; Burton, Barbara K.; Ficicioglu, Can et al.
- Monitoring and integrated care coordination of patients with alpha-mannosidosis: A global Delphi consensus study
- MOLECULAR GENETICS AND METABOLISM. Bd. 142. H. 4. 2024
- Muetze, Ulrike; Ottenberger, Alina; Gleich, Florian et al.
- Neurological outcome in long-chain hydroxy fatty acid oxidation disorders
- ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY. Bd. 11. H. 4. 2024 S. 883-898
- Reischl-Hajiabadi, Anna T.; Schnabel, Elena; Gleich, Florian et al.
- Outcomes after newborn screening for propionic and methylmalonic acidemia and homocystinurias
- JOURNAL OF INHERITED METABOLIC DISEASE. Bd. 47. H. 4. 2024 S. 674-689