Morquio Syndrome
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-03-10Clinical Trial Update: Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI [Phase 1, Phase 2]
This clinical trial update reports safety and efficacy data for Voxzogo (vosoritide) in growth deficits associated with Morquio A syndrome (…
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CLINICAL_TRIALS trial_result · 2022-12-19Clinical Trial Update: Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI [Phase 2]
Phase 2 study evaluating the effect of losartan on cardiovascular outcomes in patients with Mucopolysaccharidosis IV A and VI.
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FDA approval · 2019-12-04FDA Approved: VIMIZIM — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved a supplemental application for VIMIZIM (elosulfase alfa) from BioMarin Pharmaceutical.
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CLINICAL_TRIALS trial_result · 2019-09-26Clinical Trial Update: BMN 110 Phase 3B in Australian Patients [Phase 3]
Phase 3B trial update for BMN 110 in Australian patients with Morquio A syndrome (Mucopolysaccharidosis IVA) has completed.
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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 trial_result · 2017-05-19Clinical Trial Update: Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) [Phase 3]
Long-term efficacy and safety extension study of BMN 110 in Morquio A syndrome (MPS IVA) completed Phase 3 with results posted.
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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clinical trial evaluating dynamic gait analysis in children with Mucopolysaccharidosis type IVA (Morquio A) has been withdrawn.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 · 2016-01-12Clinical Trial Update: Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation [Phase 2]
Phase 2 efficacy and safety study of BMN 110 (elosulfase alfa) for Morquio A syndrome with limited ambulation was terminated, with results p…
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CLINICAL_TRIALS other · 2014-04-02Clinical Trial Update: BMN 110 US Expanded Access Program
Expanded access program update for BMN 110 (elosulfase alfa) in Morquio A Syndrome (MPS IVA); status indicates marketing approval.
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CLINICAL_TRIALS research · 2013-12-04Clinical Trial Update: Psychological Concomitants of Morquio Syndrome (The MAP Study)
A clinical trial update describes the MAP Study on psychological concomitants in Morquio syndrome (MPS IV), which has completed.