77 results on '"AHMED, ALIA"'
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2. Dose matters: Need for higher dose of avalglucosidase alfa in late-onset Pompe disease patients with disease progression on standard dose
3. Developing biomarker trends to establish the efficacy of treatment, and to compare relative differences between different drugs
4. Frequent GAG monitoring to evaluate the efficacy of enzyme replacement therapy in MPS IVA
5. Quantitative brain morphometry identifies cerebellar, cortical, and subcortical gray and white matter atrophy in late-onset Tay-Sachs disease
6. Disparities in late and lost: Pediatricians' role in following Pompe disease identified by newborn screening
7. Lyso-Gl3 levels in patients during transition from agalsidase beta to migalastat
8. Resolving variants of unknown significance and pseudodeficiency alleles
9. Long-term intravenous galsulfase in Maroteaux-Lamy syndrome further reduces urine glycosaminoglycans (GAG) after hematopoietic stem cell transplantation and improves cardiac function and endurance
10. How help-seeking behaviors help reduce emergency nurses' stress?
11. Quantifying medical manifestations in Hurler syndrome with the infant physical symptom score: associations with long-term physical and adaptive outcomes
12. Comparison of therapeutic potential of ERT to chaperone therapy in I270T related Fabry disease
13. Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I
14. Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I
15. Newborn screening experience and outcome from a Minnesota Pompe disease consortium
16. MPS-specific physical symptom score (PSS) and adaptive functions in MPS IVA: A cross sectional study
17. Development of mucopolysaccharidosis or MPS specific Infant Physical Symptom Score (IPSS)
18. Evidence of problems with “processing efficiency” in attenuated mucopolysaccharidosis type I
19. Report on Online Teaching and Learning Amid COVID-19
20. A longitudinal study of neurocognition and behavior in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation
21. Longitudinal study of brain volume changes in infantile and juvenile gangliosidoses
22. Observing the advanced disease course in mucopolysaccharidosis, type IIIA; a case series
23. Association of hippocampus and amygdala volumes with neurocognitive and behavioral outcomes in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation
24. Distinct progression patterns of brain disease in infantile and juvenile gangliosidoses: Volumetric quantitative MRI study
25. Long-term cognitive and somatic outcomes of enzyme replacement therapy in untransplanted Hurler syndrome
26. Career Onion: Peeling Off the Layers for Occupational Preferences and Career Aspirations
27. Neurocognitive and behavioral longitudinal trajectory of Hurler-Scheie syndrome patients with L238Q mutation
28. A longitudinal study of emotional adjustment, quality of life and adaptive function in attenuated MPS II
29. Association of somatic burden of disease with age and neuropsychological measures in attenuated mucopolysaccharidosis types I, II and VI
30. Association of physical symptom score (PSS) with age and cognitive measures in attenuated mucopolysaccharidosis types I, II and VI
31. Brain MRI patterns in MPS IIIB (Sanfilippo syndrome type B): A longitudinal study
32. Outcomes of enzyme replacement therapy in a 14-year-old female with Hurler syndrome
33. Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment
34. Quantifying behaviors of children with Sanfilippo syndrome: The Sanfilippo Behavior Rating Scale
35. Neurobehavioral outcomes in Sanfilippo syndrome type B compared to type A
36. Amygdalar volumes and acquired autistic symptoms in MPS IIIA
37. Clinical outcomes of Hurler syndrome treated exclusively with enzyme replacement therapy from a young age
38. Is somatic disease burden associated with brain disease phenotype in MPS I?
39. Brain volumes and cognitive function in MPS IIIB (Sanfilippo syndrome type B): Cross-sectional study
40. Cognitive, medical, and neuroimaging characteristics of attenuated mucopolysaccharidosis type II
41. Deregulation of miR-146a expression in a mouse model of pancreatic cancer affecting EGFR signaling
42. MPS (mucopolysaccharidosis) specific physical symptom score - development, reliability and validity
43. Brain volumes and cognition in mucopolysaccharidosis type VI
44. Neurocognitive and neuropsychiatric phenotypes associated with the mutation L238Q of the α-L-iduronidase gene in Hurler–Scheie syndrome
45. Longitudinal studies of brain structure and function in MPS disorders: a study of the lysosomal disease network
46. The MPS health assessment questionnaire: preliminary normative and validity data
47. Brain MRI abnormalities in mucopolysaccharidosis type I: cross-sectional Study
48. The development of brain and neurocognitive function in typically developing children ages 4-7 years
49. White matter structure and function in attenuated MPS II
50. Age and developmental change in adaptive behavior in severe and attenuated mucopolysaccharidosis type 1
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