Records containing: MPS IVA
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-03-17PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
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CLINICAL_TRIALS study record update · 2026-03-10A Proof of Concept Study to Evaluate the Safety and Efficacy of Voxzogo (Vosoritide) for the Treatment of Growth Deficits in MPS IVA and VI
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CLINICAL_TRIALS study record update · 2026-01-26Cardiovascular Structure and Function in Mucopolysaccharidosis Types I and IVA
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CLINICAL_TRIALS study record update · 2025-05-14Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)
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CLINICAL_TRIALS study record update · 2024-03-05A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
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CLINICAL_TRIALS study record update · 2019-10-17Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy
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CLINICAL_TRIALS study record update · 2017-07-06A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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CLINICAL_TRIALS study record update · 2017-05-19A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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CLINICAL_TRIALS study record update · 2016-02-01A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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CLINICAL_TRIALS study record update · 2016-01-12A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation
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CLINICAL_TRIALS study record update · 2015-08-13Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome Type A and Maroteaux-Lamy Syndrome
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CLINICAL_TRIALS study record update · 2014-04-02A Multicenter, Open-label BMN 110 US Expanded Access Program (BMN 110 US EAP) to Provide BMN 110 to Patients Diagnosed With MPS IVA