Molecular basis of neurocognitive dysfunction and psychosis in Alpha-Mannosidosis
Journal of Translational Genetics and Genomics
Journal of Translational Genetics and Genomics
Enzyme replacement therapy with velmanase alfa: Novel global treatment response model and outcomes in patients with alpha-mannosidosis
Molecular Genetics and Metabolism
Health Related Quality of Life, Disability, and Pain in Alpha Mannosidosis: Long-Term Data of Enzyme Replacement Therapy With Velmanase Alfa
Journal of Inborn Errors of Metabolism and Screening
Long-term safety and efficacy of velmanase alfa treatment in children under 6 years of age with alpha-mannosidosis
Journal of Inherited Metabolic Diseases
Cost-effectiveness of velmanase alfa vs. bone marrow transplantation in patients with moderate alpha-mannosidosis
Biotechnology & Biotechnological Equipment
Enzyme replacement therapy for alpha-mannosidosis: 12 months follow-up of a single centre, randomised, multiple dose study
Journal of Inherited Metabolic Diseases
Relationship between MAN2B1 genotype, antidrug antibody detection, and long-term velmanase alfa treatment outcomes
Journal of Inherited Metabolic Diseases
Long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment
Journal of Inherited Metabolic Diseases
Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis
Journal of Inherited Metabolic Diseases
Cost-effectiveness of velmanase alfa vs. bone marrow transplantation
Biotechnology & Biotechnological Equipment
α-Mannosidosis - An underdiagnosed lysosomal storage disease in individuals with an ‘MPS-like’ phenotype
Molecular Genetics and Metabolism
Recognition of alpha-mannosidosis in paediatric and adult patients
Molecular Genetics and Metabolism
White matter alteration and cerebellar atrophy are hallmarks of brain MRI in alpha-mannosidosis
Molecular Genetics and Metabolism