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?
Paul R. Harmatz
Elite in Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)
Dr. Paul R. Harmatz
Pediatric Gastroenterology | Gastroenterology
Elite in Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)
Dr. Paul R. Harmatz
Pediatric Gastroenterology | Gastroenterology

UCSF Benioff Children's Hospital Gastroenterology Clinic

744 52nd St, 5th Floor, 
Oakland, CA 
510-428-3058
Languages Spoken:
English
See accepted insurances
Offers Telehealth

Paul Harmatz is a Gastroenterologist and a Pediatric Gastroenterologist in Oakland, CA. Dr. Harmatz is rated as an Elite provider by MediFind in the treatment of Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome). His top areas of expertise are Mucopolysaccharidoses (MPS), Mucopolysaccharidosis Type 6 (MPS VI, Maroteaux-Lamy Syndrome), Mucopolysaccharidosis Type 2 (MPS II, Hunter Syndrome), and Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome).

Elite in Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)
Dr. William G. Mackenzie
Pediatric Orthopedics | Orthopedics
Elite in Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)
Dr. William G. Mackenzie
Pediatric Orthopedics | Orthopedics
1600 Rockland Rd, Nemours Dupont Pediatrics, 
Wilmington, DE 
302-651-4000
Languages Spoken:
English
See accepted insurances
Accepting New Patients

William Mackenzie is a Pediatric Orthopedics specialist and an Orthopedics provider in Wilmington, Delaware. Dr. Mackenzie is rated as an Elite provider by MediFind in the treatment of Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome). His top areas of expertise are Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome), Brachydactyly Mononen Type, Achondroplasia, Osteotomy, and Spinal Fusion. Dr. Mackenzie is currently accepting new patients.

 
 
 
 
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Elite in Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)
Amelia Morrone
Elite in Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)
Amelia Morrone
Florence, IT 

Amelia Morrone practices in Florence, Italy. Ms. Morrone is rated as an Elite expert by MediFind in the treatment of Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome). Her top areas of expertise are Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome), Brachydactyly Mononen Type, Mucopolysaccharidosis Type 4A (MPS IVA, Morquio Syndrome Type A), and PMM2-Congenital Disorder of Glycosylation.

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What are the latest Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome) Clinical Trials?
Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)
Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)
Enrollment Status: Recruiting
Publish Date: May 14, 2025
Intervention Type: Diagnostic test

Summary: Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all ages of patients to better understand the progress of their disease and the most serious clinical problems (cervical instability and stenosis, tracheal obstruction, hyperl...

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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 11, 2025
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