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Mucopolysaccharidosis IV (Morquio Syndrome)

Genetic disorder of mucopolysaccharide metabolism characterized by skeletal abnormalities, joint instability, development of cervical myelopathy, and excessive urinary keratan sulfate. There are two biochemically distinct forms, each due to a deficiency of a different enzyme.
Also Known As:
Morquio Syndrome; Morquio Disease; Eccentroosteochondrodysplasia; GALNS Deficiency; Galactosamine-6-Sulfatase Deficiency; MPS IV A; MPS IV B; MPS IVA; MPS IVB; Morquio A Disease; Morquio B Syndrome; Morquio Syndrome A; Morquio Syndrome B; Morquio Syndrome, Type B; Morquio's Disease Type B; Morquio's Syndrome; Morquio-B Disease; Mucopolysaccharidosis 4; Mucopolysaccharidosis 4B; Mucopolysaccharidosis Type IV; Mucopolysaccharidosis Type IV A; Mucopolysaccharidosis Type IV B; Mucopolysaccharidosis Type IVA; Mucopolysaccharidosis Type IVB; Deficiencies, GALNS; Deficiencies, Galactosamine-6-Sulfatase; Deficiency, GALNS; Deficiency, Galactosamine-6-Sulfatase; Disease, Morquio; Disease, Morquio A; Disease, Morquio's; Disease, Morquio-B; Eccentro Osteochondrodysplasia; Eccentro-Osteochondrodysplasias; Eccentroosteochondrodysplasias; GALNS Deficiencies; Galactosamine 6 Sulfatase Deficiency; Galactosamine-6-Sulfatase Deficiencies; IV, Mucopolysaccharidosis Type; IVs, Mucopolysaccharidosis Type; Morquio B Disease; Morquio Syndromes; Morquios Disease; Morquios Syndrome; Mucopolysaccharidosis 4Bs; Mucopolysaccharidosis Type IVBs; Mucopolysaccharidosis Type IVs; Syndrome A, Morquio; Syndrome, Morquio; Syndrome, Morquio B; Syndrome, Morquio's; Syndromes, Morquio; Type IV, Mucopolysaccharidosis; Type IVs, Mucopolysaccharidosis; Eccentro-Osteochondrodysplasia; Morquio's Disease
Networked: 395 relevant articles (20 outcomes, 39 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1. Mucopolysaccharidoses
2. Hypophosphatasia
3. Mucopolysaccharidosis IV (Morquio Syndrome)
4. GM1 Gangliosidosis (Gangliosidosis GM1)
5. Lysosomal Storage Diseases (Lysosomal Storage Disease)

Experts

1. Tomatsu, Shunji: 49 articles (11/2021 - 07/2003)
2. Orii, Tadao: 30 articles (06/2019 - 07/2003)
3. Montaño, Adriana M: 20 articles (01/2022 - 11/2005)
4. Giugliani, Roberto: 20 articles (02/2019 - 01/2004)
5. Suzuki, Yasuyuki: 17 articles (11/2021 - 04/2004)
6. Yamaguchi, Seiji: 16 articles (07/2021 - 12/2003)
7. Mackenzie, William G: 13 articles (06/2021 - 09/2012)
8. Mason, Robert W: 13 articles (06/2019 - 12/2012)
9. Alméciga-Díaz, Carlos J: 12 articles (07/2020 - 09/2008)
10. Barrera, Luis A: 11 articles (01/2020 - 01/2004)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Mucopolysaccharidosis IV:
1. human GALNS proteinIBA
2. EnzymesIBA
3. Keratan SulfateIBA
4. Biomarkers (Surrogate Marker)IBA
5. SulfatasesIBA
6. N-acetylgalactosamine 6-sulfateIBA
7. Ezetimibe (Zetia)FDA Link
8. pranlukastIBA
9. Proteins (Proteins, Gene)FDA Link
10. beta-Galactosidase (Lactaid)IBA

Therapies and Procedures

1. Enzyme Replacement Therapy
01/01/2020 - "Enzyme replacement therapies (ERTs) are now available for several MPS diseases (MPS I, MPS II, MPS IVA, MPS VI, and MPS VII), but assessment of the efficacy of treatment can be challenging because these are rare, progressive, and highly heterogeneous diseases; because some clinical manifestations may be irreversible if treatment initiation is delayed; and because determining the benefits of a treatment to prevent those manifestations may take prolonged periods of time. "
11/01/2014 - "Our observation is clinically significant because patients with Morquio syndrome are often diagnosed only after they develop typical skeletal manifestations, which reduces the efficacy of available enzyme replacement therapy, so the umbilical sign we report here can facilitate a much earlier diagnosis. "
10/01/2023 - "This study aimed to characterize the phenotype and genotype from patients with MPS IV-A, who are under Enzyme Replacement Therapy at the Children's Neuropsychiatry Service of the Hospital Clínico San Borja Arriarán, Santiago, Chile, and to determine if there is any association between genotype and phenotype with those findings. "
01/01/2023 - "Inflammatory process and oxidative/nitrative stress: in vivo study in mucopolysaccharidosis type IV A patients under long-term enzyme replacement therapy."
09/01/2019 - "The aim of this study was to characterise and catalogue the long-term respiratory changes in patients with MPS IVA treated with, or without, enzyme replacement therapy (ERT). "
2. Activities of Daily Living (ADL)
3. Therapeutics
4. Pedicle Screws
5. Bone Marrow Transplantation (Transplantation, Bone Marrow)