Mucopolysaccharidosis type 4
Therapeutic area
Metabolic Disease
Rare disease
Yes
- 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
- 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
- All therapeutic areas
- Metabolic Disease
- Severity
- Severe
- Developmental
- Genetic
- Hereditary
- Inborn Error of Metabolism
- Syndromic
- MONDO ID
- 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