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Topics

Learn about Mucopolysaccharidosis topics

Safety and Efficacy Profile

Publication

Topics

Safety and Efficacy Profile

Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS)

Long-term enzyme replacement therapy: Findings from the mucopolysaccharidosis VI clinical surveillance program after 15 years follow-up

Long-term outcomes of MPS IVA patients treated with elosulfase alfa: Findings from the Morquio A Registry Study (MARS) after 10 years

Enzyme replacement therapy is associated with improved endurance in MPS IVA patients at different ages

Real-World Impact of Enzyme Replacement Therapy on Endurance in Patients with MPS IVA

Long-term outcomes of MPS IVA patients treated with elosulfase alfa: Findings from the Morquio A Registry Study (MARS) after 6 years

Long-Term Outcomes in Adults with MPS IVA Treated with Elosulfase Alfa: Findings from the Morquio A Registry Study

Genetic Testing & Diagnosis

Skeletal dysplasia gene panel with integrated enzyme follow-up for the diagnosis of lysosomal storage disorders: MPS IVA case series

Gene panels for skeletal dysplasia and epilepsy: Maximizing clinical utility through careful design, regular review, and clinician-laboratory collaboration

Clinical utility of a sponsored, no-charge skeletal dysplasia gene panel testing program: Results from >2600 tests

Clinical Utility of a Sponsored, No-cost Skeletal Dysplasia Gene Panel Testing Program: Results from 850 Tests

Molecular Basis of Mucopolysaccharidosis IVA (Morquio A Syndrome): A Review and Classification of GALNS Gene Variants and Reporting of New Variants

Real World Evidence