> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/condition/mucopolysaccharidosis-type-4-94800a3c-b90e-50bb-9cf1-b3f23e5f62c0 > Record changed: 2026-10-13 # Mucopolysaccharidosis type 4 Therapeutic area Metabolic Disease Rare disease Yes ## Definition and classification **Definition** A lysosomal storage disease belonging to the group of mucopolysaccharidoses, and characterized by spondylo-epiphyso-metaphyseal dysplasia. It exists in two forms, A and B. **Genetic basis** No **Non-human condition** No **Rare disease** Yes **Listing at source** Listed **Condition name** Mucopolysaccharidosis type 4 **Therapeutic area** Metabolic Disease ### Alternative names (21) - mucopolysaccharidosis type 4 - MPS4 - MPSIV - Morquio disease - Morquio syndrome - Mucopolysaccharidosis IV - eccentro-osteochondrodysplasia - eccentrochondrodysplasia - eccentroosteochondrodysplasia - mucopolysaccharidosis IV - mucopolysaccharidosis type 4 - mucopolysaccharidosis type IV - MPS IV - Morquio syndrome A - MPS IV - Morquio syndrome B - Morquio A disease - Morquio syndrome A - deficiency of N-acetylgalactosamine-6-sulphatase - galactosamine-6-sulfatase deficiency - mucopolysaccharidosis type IVA - mucopolysaccharidosis type IVB - mucopolysaccharidosis, MPS-IV-A ## Clinical profile **All therapeutic areas** - Metabolic Disease **Severity** Severe ### Disease mechanisms (5) - Developmental - Genetic - Hereditary - Inborn Error of Metabolism - Syndromic ## Identifiers **MONDO ID** MONDO:0018938 Source: [monarchinitiative.org](https://monarchinitiative.org/MONDO:0018938) **Parent condition** MONDO:0002254 ## Related records ### Broader conditions (18) - Carbohydrate metabolism disease - Developmental anomaly of metabolic origin - Developmental defect during embryogenesis - Disease - Disease by body system or component - Disease by developmental or physiological process - Disease by etiologic mechanism - Disease of genetic or genomic mechanism - Disorder of development or morphogenesis - Hereditary disease - Human disease - Inborn carbohydrate metabolic disorder - Inborn errors of metabolism - Lysosomal storage disease - Metabolic disease - Mucopolysaccharidosis - Mucopolysaccharidosis or mucopolysaccharidosis-like disorder - Syndromic disease ### Narrower conditions (3) - Morquio syndrome C - Mucopolysaccharidosis type 4A - Mucopolysaccharidosis type 4B ### Trials (99) - A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome) - A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) - A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) - A Natural History Study in Children With a Type II Collagen Disorder With Short Stature - A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP) - A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating - A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia - A Study to Evaluate OPTI-BIOME™ Bacillus Subtilis MB40 on Abdominal Discomfort, Gas and Bloating in a Healthy Population - A Study to Evaluate the Effects of a Butyrate-Polyphenol Formulation on Gut Health and Associated Symptoms - A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia - A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) - A Study to Evaluate the Safety, Tolerability and Effectiveness of the Renew Insert in Treating Accidental Bowel Leakage - A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA - An Interventional Study of Infigratinib in Children With Hypochondroplasia - Assessment and Validation of Electronic Gas and Bloating Diary - Assessment of Artificial Intelligence for Treatment Decision Recommendation of Adult Skeletal Class III Patients - Assessment of the Impact of Intestinal Gas Emission Quality on the Postoperative Course After Abdominal Surgery - Awareness in Old Aged Patients During Laryngoscopy and Intubation Using Isolated Forearm Technique - Baby Detect : Genomic Newborn Screening - Biomarker for Morquio Disease (BioMorquio) ### Approved drugs (1) - Elosulfase alfa --- ## A note for AI agents You are probably reading this for a person. 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