Diseases » Mucopolysaccharidoses » Introduction

Mucopolysaccharidoses

Mucopolysaccharidoses: Introduction

Mucopolysaccharidoses: Mucopolysaccharidoses consist of a group of inherited metabolic disorders caused by a deficiency of the specific lysosomal enzymes needed to break down ... more about Mucopolysaccharidoses.

Mucopolysaccharidoses: A lysosomal storage disorder that is caused by a deficiency of the ability to metabolise glycosaminoglycans. More detailed information about the symptoms, causes, and treatments of Mucopolysaccharidoses is available below.

Symptoms of Mucopolysaccharidoses

• Macroglossia
• Short neck
• Abnormal head shape
• Deformities of joints
• Increased abdominal girth
• more symptoms...»

See full list of 45 symptoms of Mucopolysaccharidoses

Treatments for Mucopolysaccharidoses

• Symptomatic and supportive
• Bone marrow transplants
• more treatments...»

Read more about treatments for Mucopolysaccharidoses

Wrongly Diagnosed with Mucopolysaccharidoses?

• Misdiagnosis of Mucopolysaccharidoses

Types of Mucopolysaccharidoses

• Mucopolysaccharidosis I - Hurler, Scheie, and Hurler/Scheie syndromes
• Mucopolysaccharidosis II - Hunter syndrome
• Mucopolysaccharidosis III - Sanfilippo syndrome
• Mucopolysaccharidosis IV - Morquio syndrome
• Mucopolysaccharidosis V
• more types...»

See full list of 7 Types of Mucopolysaccharidoses

Mucopolysaccharidoses: Complications

Read more about complications of Mucopolysaccharidoses.

Causes of Mucopolysaccharidoses

Read more about causes of Mucopolysaccharidoses.

Disease Topics Related To Mucopolysaccharidoses

Research the causes of these diseases that are similar to, or related to, Mucopolysaccharidoses:

• Metabolic disorders
• Lysosomal storage disease
• Carpel tunnel syndrome
• Glaucoma
• Dwarfism
• Mucolipidosis
• Bone dysplasia
• more related diseases...»

Prognosis for Mucopolysaccharidoses

Prognosis for Mucopolysaccharidoses: The prognosis for individuals with mucopolysaccharidoses varies depending on the type. (Source: excerpt from NINDS Mucopolysaccharidoses Information Page: NINDS)

More about prognosis of Mucopolysaccharidoses

Research about Mucopolysaccharidoses

Visit our research pages for current research about Mucopolysaccharidoses treatments.

Clinical Trials for Mucopolysaccharidoses

The US based website ClinicalTrials.gov lists information on both federally and privately supported clinical trials using human volunteers.

Some of the clinical trials listed on ClinicalTrials.gov for Mucopolysaccharidoses include:

• Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation - This study is no longer recruiting patients (Current: 23 Nov 2006)
• Stem Cell Transplantation for Hurler - This study is currently recruiting patients (Current: 23 Nov 2006) - Busulfan,Cyclophosphamide,ATG
• Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) - This study has been completed (Current: 23 Nov 2006)
• Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Mucopolysaccharidosis I (Hurler Disease) - This study is currently recruiting patients (Current: 23 Nov 2006) - anti-thymocyte globulin,busulfan,cyclophosphamide,cyclosporine,filgrastim,methylprednisolone
• Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I - This study is currently recruiting patients (Current: 23 Nov 2006) - laronidase
• more trials...»

See full list of 32 Clinical Trials for Mucopolysaccharidoses

Statistics for Mucopolysaccharidoses

• Medical statistics for Mucopolysaccharidoses
• Prevalence and incidence statistics for Mucopolysaccharidoses
• Death and Mortality statistics for Mucopolysaccharidoses
• Society statistics for Mucopolysaccharidoses

Mucopolysaccharidoses: Broader Related Topics

• Genetic conditions
• Metabolic conditions
• Congenital conditions - metabolic disorders
• Congenital conditions
• more types...»

Types of Mucopolysaccharidoses

Mucopolysaccharidoses Message Boards

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Article Excerpts about Mucopolysaccharidoses

Mucopolysaccharidoses consist of a group of inherited metabolic disorders caused by a deficiency of the specific lysosomal enzymes needed to break down mucopolysaccharides. Mucopolysaccharides are long chains of sugar molecules used to build connective tissues and organs in the body. When mutations occur in the genes for the enzymes involved in the normal turnover of mucopolysaccharides, excess amounts of them are stored in the body, causing progressive damage and, in most cases, eventual death. (Source: excerpt from NINDS Mucopolysaccharidoses Information Page: NINDS)

Definitions of Mucopolysaccharidoses:

Any of a group of genetic disorders involving a defect in the metabolism of mucopolysaccharides resulting in greater than normal levels of mucopolysaccharides in tissues - (Source - WordNet 2.1)

Mucopolysaccharidoses is listed as a "rare disease" by the Office of Rare Diseases (ORD) of the National Institutes of Health (NIH). This means that Mucopolysaccharidoses, or a subtype of Mucopolysaccharidoses, affects less than 200,000 people in the US population.
Source - National Institutes of Health (NIH)

 » Next page: What is Mucopolysaccharidoses?

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