Mucopolysaccharidoses (MPS) Overview

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Learn About Mucopolysaccharidoses (MPS)

What is the definition of Mucopolysaccharidoses (MPS)?

Mucopolysaccharidoses (MPSs) are a group of rare diseases in which the body is missing or does not have enough of an enzyme needed to break down long chains of sugar molecules. These chains of molecules are called glycosaminoglycans (formerly called mucopolysaccharides). As a result, the molecules build up in different parts of the body and cause various health problems. There are several types of MPSs, including:

  • MPS I (Hurler syndrome; Hurler-Scheie syndrome; Scheie syndrome)
  • MPS II (Hunter syndrome)
  • MPS III (Sanfilippo syndrome)
  • MPS IV (Morquio syndrome)
What are the alternative names for Mucopolysaccharidoses (MPS)?

MPS; Lysosomal storage disease - mucopolysaccharidosis

Who are the top Mucopolysaccharidoses (MPS) Local Doctors?

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What are the latest Mucopolysaccharidoses (MPS) Clinical Trials?
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie
Enrollment Status: Recruiting
Publish Date: February 26, 2025
Intervention Type: Biological
Study Phase: Phase 1

Summary: A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.

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A Multi-center, Randomized, Active Controlled Clinical Trial to Evaluate the Efficacy and Safety of OTL-203 in Subjects with Mucopolysaccharidosis Type I, Hurler Syndrome (MPS-IH) Compared to Standard of Care with Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT)
A Multi-center, Randomized, Active Controlled Clinical Trial to Evaluate the Efficacy and Safety of OTL-203 in Subjects with Mucopolysaccharidosis Type I, Hurler Syndrome (MPS-IH) Compared to Standard of Care with Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT)
Enrollment Status: Recruiting
Publish Date: February 25, 2025
Intervention Type: Genetic
Study Phase: Phase 3

Summary: A multi-center randomized clinical trial to compare OTL-203 (gene therapy) with stem cell transplant (standard of care) in patients with MPS-IH (Hurler syndrome).

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Who are the sources who wrote this article ?

Published Date: April 24, 2023
Published By: Anna C. Edens Hurst, MD, MS, Associate Professor in Medical Genetics, The University of Alabama at Birmingham, Birmingham, AL. Review provided by VeriMed Healthcare Network. Also reviewed by David C. Dugdale, MD, Medical Director, Brenda Conaway, Editorial Director, and the A.D.A.M. Editorial team.

What are the references for this article ?

Pyeritz RE. Inherited diseases of connective tissue. In: Goldman L, Cooney KA, eds. Goldman-Cecil Medicine. 27th ed. Philadelphia, PA: Elsevier; 2024:chap 239.

Spranger JW. Mucopolysaccharidoses. In: Kliegman RM, St. Geme JW, Blum NJ, Shah SS, Tasker RC, Wilson KM, eds. Nelson Textbook of Pediatrics. 21st ed. Philadelphia, PA: Elsevier; 2020:chap 107.

Turnpenny PD, Ellard S, Cleaver R. Inborn errors of metabolism. In: Turnpenny PD, Ellard S, Cleaver R, eds. Emery's Elements of Medical Genetics and Genomics. 16th ed. Philadelphia, PA: Elsevier; 2022:chap 18.