Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)
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Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome) Overview

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Learn About Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)

View Main Condition: Mucopolysaccharidoses (MPS)

What is the definition of Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)?
Mucopolysaccharidosis type IV (MPS IV), also known as Morquio syndrome, is a rare metabolic condition in which the body is unable to break down long chains of sugar molecules called glycosaminoglycans. As a result, toxic levels of these sugars accumulate in cell structures called lysosomes, leading to the various signs and symptoms associated with the condition. These signs and symptoms may include abnormalities of the skeleton, eyes, heart and respiratory system. There are two forms of MPS IV. MPS IVA is caused by changes in the GALNS gene. MPS IVB is caused by genetic changes in the GLB1 gene. Both forms are inherited in an autosomal recessive manner.
What are the alternative names for Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)?
  • Mucopolysaccharidosis type IV
  • MPS4
  • MPSIV
  • Morquio disease
  • Mucopolysaccharidosis type 4
What are the different types of Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)?

Common conditions include: Mucopolysaccharidosis Type 4A (MPS IVA, Morquio Syndrome Type A)

Who are the top Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome) Local Doctors?
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What are the latest Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome) Clinical Trials?
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Enrollment Status: Recruiting
Publish Date: April 08, 2026
Intervention Type: Other

Summary: This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.

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PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Who is this study for: Pregnant women age 18 to 50 carrying fetuses between 18 to 34 weeks gestation diagnosed with lysosomal storage disorders
Enrollment Status: Recruiting
Publish Date: March 17, 2026
Intervention Type: Drug
Study Drug: Aldurazyme (laronidase)
Study Phase: Phase 1

Summary: For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

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Who are the sources who wrote this article ?

Published Date: May 02, 2022
Published By: Genetic and Rare Diseases Informnation Center