Mucopolysaccharidosis

Common Name(s)

Mucopolysaccharidosis

Description for this condition is not yet available.
 

Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Mucopolysaccharidosis" for support, advocacy or research.

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Canadian Society for Mucopolysaccharide and Related Diseases Inc.

The Canadian MPS Society is committed to providing support to families affected with MPS and related diseases, educating medical professionals and the general public about MPS and related diseases, and raising funds for research so that one day cures can be found for all types of MPS and related diseases.

Last Updated: 24 Nov 2009

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CNS Foundation

CNS Foundation's Mission is Three-fold: - Expedite scientific research to find treatments and cures for the more than 14 million children living with some form of neurological disorder - Provide families and health care professionals with up-to-date information and resources on the latest discoveries in pediatric neurology - Educate the public, and public officials, on the critical importance of funding pediatric neurological disorder research

Last Updated: 12 Oct 2013

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National Endowment for Alzheimer's Research

At NEAR, our mission is to investigate the underlying causes of Alzheimer's and to develop rapid clinical interventions both for Alzheimer's and other brain disorders. A secondary mission is to promote public awareness and education with respect to Alzheimer's disease and related dementia, and to encourage the involvement of individuals, businesses, foundations, and government in finding a cure.

Last Updated: 20 Dec 2012

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General Support Organizations

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Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Mucopolysaccharidosis" for support, advocacy or research.

Logo
Canadian Society for Mucopolysaccharide and Related Diseases Inc.

The Canadian MPS Society is committed to providing support to families affected with MPS and related diseases, educating medical professionals and the general public about MPS and related diseases, and raising funds for research so that one day cures can be found for all types of MPS and related diseases.

http://www.mpssociety.ca

Last Updated: 24 Nov 2009

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CNS Foundation

CNS Foundation's Mission is Three-fold: - Expedite scientific research to find treatments and cures for the more than 14 million children living with some form of neurological disorder - Provide families and health care professionals with up-to-date information and resources on the latest discoveries in pediatric neurology - Educate the public, and public officials, on the critical importance of funding pediatric neurological disorder research

http://www.cnsfoundation.org

Last Updated: 12 Oct 2013

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National Endowment for Alzheimer's Research

At NEAR, our mission is to investigate the underlying causes of Alzheimer's and to develop rapid clinical interventions both for Alzheimer's and other brain disorders. A secondary mission is to promote public awareness and education with respect to Alzheimer's disease and related dementia, and to encourage the involvement of individuals, businesses, foundations, and government in finding a cure.

http://www.memorymatters.org

Last Updated: 20 Dec 2012

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Scientific Literature

Articles from the PubMed Database

Research articles describe the outcome of a single study. They are the published results of original research.
The terms "Mucopolysaccharidosis" returned 258 free, full-text research articles on human participants. First 3 results:

Mesenchymal stem cells do not prevent antibody responses against human α-L-iduronidase when used to treat mucopolysaccharidosis type I.
 

Author(s): Priscila Keiko Matsumoto Martin, Roberta Sessa Stilhano, Vivian Yochiko Samoto, Christina Maeda Takiya, Giovani Bravin Peres, Yara Maria Correa da Silva Michelacci, Flavia Helena da Silva, Vanessa Gonçalves Pereira, Vânia D'Almeida, Fabio Luiz Navarro Marques, Andreia Hanada Otake, Roger Chammas, Sang Won Han

Journal:

 

Mucopolysaccharidosis type I (MPSI) is an autosomal recessive disease that leads to systemic lysosomal storage, which is caused by the absence of α-L-iduronidase (IDUA). Enzyme replacement therapy is recognized as the best therapeutic option for MPSI; however, high titers of anti-IDUA ...

Last Updated: 19 Mar 2014

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Impact of enzyme replacement therapy on linear growth in Korean patients with mucopolysaccharidosis type II (Hunter syndrome).
 

Author(s): Sung Yoon Cho, Rimm Huh, Mi Sun Chang, Jieun Lee, Younghee Kwun, Se Hyun Maeng, Su Jin Kim, Young Bae Sohn, Sung Won Park, Eun-Kyung Kwon, Sun Ju Han, Jooyoun Jung, Dong-Kyu Jin

Journal: J. Korean Med. Sci.. 2014 Feb;29(2):254-60.

 

Hunter syndrome (or mucopolysaccharidosis type II [MPS II]) arises because of a deficiency in the lysosomal enzyme iduronate-2-sulfatase. Short stature is a prominent and consistent feature in MPS II. Enzyme replacement therapy (ERT) with idursulfase (Elaprase®) or idursulfase beta ...

Last Updated: 19 Feb 2014

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Natural history and clinical assessment of Taiwanese patients with mucopolysaccharidosis IVA.
 

Author(s): Hsiang-Yu Lin, Chih-Kuang Chuang, Ming-Ren Chen, Pao Chin Chiu, Yu-Yuan Ke, Dau-Ming Niu, Fuu-Jen Tsai, Wuh-Liang Hwu, Ju-Li Lin, Shuan-Pei Lin

Journal:

 

Mucopolysaccharidosis IVA (MPS IVA) is a rare lysosomal storage disorder caused by N-acetylgalactosamine-6-sulfatase deficiency, which catalyzes a step in the catabolism of glycosaminoglycans, keratan sulfate and chondroitin-6-sulfate. This disease has a variable age of onset and ...

Last Updated: 17 Feb 2014

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Reviews from the PubMed Database

Review articles summarize what is currently known about a disease. They discuss research previously published by others.
The terms "Mucopolysaccharidosis" returned 24 free, full-text review articles on human participants. First 3 results:

Attenuated osteoarticular phenotype of type VI mucopolysaccharidosis: a report of four patients and a review of the literature.
 

Author(s): Agnieszka Jurecka, Ekaterina Zakharova, Vera Malinova, Elena Voskoboeva, Anna Tylki-Szymańska

Journal: Clin. Rheumatol.. 2014 May;33(5):725-31.

 

Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome, MPS VI, OMIM 253200) is caused by mutations in the gene coding for N-acetylgalactosamine-4-sulfatase (4-sulfatase, arylsulfatase B, ARSB, EC 3.1.6.12), a lysosomal enzyme involved in the degradation of dermatan sulfate (DS). ...

Last Updated: 28 Apr 2014

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Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure.
 

Author(s): Eveline J Langereis, Andrea Borgo, Ellen Crushell, Paul R Harmatz, Peter M van Hasselt, Simon A Jones, Paula M Kelly, Christina Lampe, Johanna H van der Lee, Thierry Odent, Ralph Sakkers, Maurizio Scarpa, Matthias U Schafroth, Peter A Struijs, Vassili Valayannopoulos, Klane K White, Frits A Wijburg

Journal:

 

Mucopolysaccharidosis type I (MPS-I) is a lysosomal storage disorder characterized by progressive multi-organ disease. The standard of care for patients with the severe phenotype (Hurler syndrome, MPS I-H) is early hematopoietic stem cell transplantation (HSCT). However, skeletal ...

Last Updated: 15 Nov 2013

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Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA.
 

Author(s): C J Hendriksz, P Harmatz, M Beck, S Jones, T Wood, R Lachman, C G Gravance, T Orii, S Tomatsu

Journal: Mol. Genet. Metab.. ;110(1-2):54-64.

 

Mucopolysaccharidosis type IVA (MPS IVA) was described in 1929 by Luis Morquio from Uruguay and James Brailsford from England, and was later found as an autosomal recessive lysosomal storage disease. MPS IVA is caused by mutations in the gene encoding the enzyme, N-acetylgalactosamine-6-sulfate ...

Last Updated: 26 Aug 2013

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Symptoms, Diagnosis, and Treatment

There are currently no related results available in GeneReviews.

 
 
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Clinical Trial Information This information is provided by ClinicalTrials.gov

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)
 

Status: Recruiting

Condition Summary: Mucopolysaccharidosis; Mucopolysaccharidosis I; Mucopolysaccharidosis II; Mucopolysaccharidosis VI

 

Last Updated: 10 Dec 2014

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Natural History Studies of Mucopolysaccharidosis III
 

Status: Recruiting

Condition Summary: Mucopolysaccharidosis Type IIIA; Mucopolysaccharidosis Type IIIB

 

Last Updated: 15 Jan 2014

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Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
 

Status: Recruiting

Condition Summary: Mucopolysaccharidosis IV A; Mucopolysaccharidosis VI

 

Last Updated: 2 Aug 2013

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