Mucopolysaccharidosis Type Iva
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CLINICAL_TRIALS research · 2026-01-26Clinical Trial Update: Cardiac Structure and Function in MPS
This clinical trial update shows ongoing recruitment for a study evaluating cardiac structure and function in patients with mucopolysacchari…
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FDA label_change · 2025-10-30FDA Approved: VIMIZIM — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for VIMIZIM (elosulfase alfa) from BioMarin Pharmaceutical, updating its labeling.
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CLINICAL_TRIALS research · 2025-05-14Clinical Trial Update: Non-invasive Functional Assessment and Pathogenesis of Morquio A
This clinical trial update describes an ongoing study evaluating non-invasive functional assessments and the pathogenesis of Morquio A (MPS …
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FDA approval · 2024-09-12FDA Approved: NAGLAZYME — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved a supplemental application for NAGLAZYME (galsulfase) from BioMarin Pharmaceutical, expanding/adjusting its approved labeling.
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CLINICAL_TRIALS trial_result · 2018-04-11Clinical Trial Update: Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
A completed MAPLE study reports longitudinal findings on the psychological effects of enzyme replacement therapy in Morquio A syndrome (MPS …
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CLINICAL_TRIALS withdrawal · 2016-07-28Clinical Trial Update: Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
The clinical trial evaluating dynamic gait analysis in children with Mucopolysaccharidosis type IVA (Morquio A) has been withdrawn.
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CLINICAL_TRIALS trial_result · 2016-02-01Clinical Trial Update: Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome [Phase 2]
Phase 2 safety study of two doses of BMN 110 for Morquio A Syndrome has been terminated with results posted.
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CLINICAL_TRIALS trial_result · 2014-12-08Clinical Trial Update: A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
This clinical trial update indicates that a BioMarin-sponsored study assessing subjects with Morquio syndrome (MPS IVA) has been terminated.
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CLINICAL_TRIALS research · 2014-09-04Clinical Trial Update: Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
This clinical trial update describes efforts to identify new biomarkers to monitor disease progression in Morquio A syndrome (Mucopolysaccha…
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CLINICAL_TRIALS trial_result · 2014-06-30Clinical Trial Update: A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA [Phase 1, Phase 2]
Phase 1/2 study evaluating safety, tolerability and efficacy of BMN-110 in people with Mucopolysaccharidosis type IVA (Morquio A) with resul…
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CLINICAL_TRIALS trial_result · 2014-04-15Clinical Trial Update: A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) [Phase 3]
Phase 3 double-blind study evaluating BMN 110 in Morquio A syndrome (MPS IVA); the trial is completed and results were posted in 2014.
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FDA approval · 2014-02-14FDA Approved: VIMIZIM — Original Application (BioMarin Pharmaceutical Inc.)
FDA approves BioMarin's Vimizim (elosulfase alfa) under an Original Biologics License Application for the treatment of Morquio A syndrome (M…
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CLINICAL_TRIALS trial_result · 2008-10-20Clinical Trial Update: Morquio's Syndrome: a Case Study [Phase 4]
Phase 4 clinical trial update for Morquio's disease (Mucopolysaccharidosis types IVA and IVB) has been terminated; the case study was sponso…