20 results on '"van Hasselt, Peter"'
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2. Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)
3. Increasing the dose of oral vitamin K prophylaxis and its effect on bleeding risk
4. IQSEC2-related encephalopathy in males and females: a comparative study including 37 novel patients
5. Aminoacyl-tRNA synthetase deficiencies in search of common themes
6. Prime editing for functional repair in patient-derived disease models
7. Timing of cognitive decline in CLN3 disease
8. Correction: Aminoacyl-tRNA synthetase deficiencies in search of common themes
9. Synapse alterations precede neuronal damage and storage pathology in a human cerebral organoid model of CLN3-juvenile neuronal ceroid lipofuscinosis
10. Emotional and behavioral problems, quality of life and metabolic control in NTBC-treated Tyrosinemia type 1 patients
11. Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: results of an international consensus procedure
12. Correction: IQSEC2-related encephalopathy in males and females: a comparative study including 37 novel patients
13. ALG6-CDG: a recognizable phenotype with epilepsy, proximal muscle weakness, ataxia and behavioral and limb anomalies
14. A Mixed Micelle Formulation for Oral Delivery of Vitamin K
15. Pain: a prevalent feature in patients with mucopolysaccharidosis. Results of a cross-sectional national survey
16. De novo mutations in beta-catenin (CTNNB1) appear to be a frequent cause of intellectual disability: expanding the mutational and clinical spectrum
17. Intracranial bleeding due to vitamin K deficiency: advantages of using a pediatric intensive care registry
18. Multiple oxidative phosphorylation deficiencies in severe childhood multi-system disorders due to polymerase gamma (POLG1) mutations
19. Erratum to: ALG6-CDG: a recognizable phenotype with epilepsy, proximal muscle weakness, ataxia and behavioral and limb anomalies
20. Transparantie
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