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

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.
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What are the alternative names for Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)?
  • Mucopolysaccharidosis type IV
  • MPS4
  • MPSIV
  • Morquio disease
  • Mucopolysaccharidosis type 4
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What are the latest Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome) Clinical Trials?
Eight At One Stroke: Attention Gangliosidoses A Registry Study for Patients With Gangliosidoses
Summary: The clinical project Eight At One Stroke: Attention Gangliosidoses represents a clinical registry for recording the clinical manifestation and the disease progression of gangliosidoses. The intention of this project is to better understand the manifestation and progression of gangliosidoses and to raise awareness of these disorders in the public health service. The patients or their families, resp...
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Biomarker for Morquio Disease AN INTERNATIONAL, MULTICENTER, EPIDEMIOLOGICAL PROTOCOL
Summary: Development of a new MS-based biomarker for the early and sensitive diagnosis of Morquio disease from plasma
Who are the sources who wrote this article ?

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

What are the Latest Advances for Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome)?
Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome.
Summary: Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome.
Understanding bias when estimating life expectancy from age at death: a simulation approach applied to Morquio syndrome A.
Summary: Understanding bias when estimating life expectancy from age at death: a simulation approach applied to Morquio syndrome A.
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Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England.
Summary: Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England.