Morquio Syndrome

Common Name(s)

Morquio Syndrome

Description for this condition is not yet available.
 

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Advocacy and Support Organizations

 

Condition Specific Organizations

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

There are currently no organizations listed in Disease InfoSearch that support this condition. Create a listing.

 

 

General Support Organizations

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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 "Morquio Syndrome" returned 26 free, full-text research articles on human participants. First 3 results:

Pharmacokinetic and pharmacodynamic evaluation of elosulfase alfa, an enzyme replacement therapy in patients with Morquio A syndrome.
 

Author(s): Yulan Qi, Donald G Musson, Becky Schweighardt, Troy Tompkins, Lynne Jesaitis, Adam J Shaywitz, Ke Yang, Charles A O'Neill

Journal: Clin Pharmacokinet. 2014 Dec;53(12):1137-47.

 

Morquio A syndrome (mucopolysaccharidosis IVA; MPS IVA) is a lysosomal storage disorder caused by deficiency of N-acetylgalactosamine-6-sulfatase, an enzyme required for degradation of the glycosaminoglycan keratan sulfate. Enzyme replacement therapy with elosulfase alfa provides ...

Last Updated: 25 Nov 2014

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Burden of disease in patients with Morquio A syndrome: results from an international patient-reported outcomes survey.
 

Author(s): Christian J Hendriksz, Christine Lavery, Mahmut Coker, Sema Kalkan Ucar, Mohit Jain, Lisa Bell, Christina Lampe

Journal:

 

Morquio A syndrome (or mucopolysaccharidosis IVa) is an ultra-rare multi-organ disease, resulting in significantly impaired functional capacity, mobility and quality of life (QoL).

Last Updated: 12 Mar 2014

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Mucopolysaccharidosis IVA (Morquio A syndrome) and VI (Maroteaux-Lamy syndrome): under-recognized and challenging to diagnose.
 

Author(s): Ralph S Lachman, Barbara K Burton, Lorne A Clarke, Scott Hoffinger, Shiro Ikegawa, Dong-Kyu Jin, Hiroki Kano, Ok-Hwa Kim, Christina Lampe, Nancy J Mendelsohn, Renée Shediac, Pranoot Tanpaiboon, Klane K White

Journal: Skeletal Radiol.. 2014 Mar;43(3):359-69.

 

Mucopolysaccharidosis IVA (MPS IVA, or Morquio A syndrome) and VI (MPS VI, or Maroteaux-Lamy syndrome) are autosomal recessive lysosomal storage disorders. Skeletal abnormalities are common initial presenting symptoms and, when recognized early, may facilitate timely diagnosis and ...

Last Updated: 27 Jan 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 "Morquio Syndrome" returned 5 free, full-text review articles on human participants. First 3 results:

Diagnostic evaluation, monitoring, and perioperative management of spinal cord compression in patients with Morquio syndrome.
 

Author(s): Joel Charrow, Tord D Alden, Catherine Ann R Breathnach, Geoffrey P Frawley, Christian J Hendriksz, Bianca Link, William G Mackenzie, Renzo Manara, Amaka C Offiah, Martha L Solano, Mary Theroux

Journal: Mol. Genet. Metab.. 2015 Jan;114(1):11-8.

 

Mucopolysaccharidosis IVA is an autosomal recessive condition caused by mutations in the GALNS gene, which encodes N-acetylgalactosamine-6-sulfatase, also called galactosamine-6-sulfatase (GALNS). A reduction in or absence of effective GALNS leads to faulty catabolism of keratan sulfate ...

Last Updated: 27 Dec 2014

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International guidelines for the management and treatment of Morquio A syndrome.
 

Author(s): Christian J Hendriksz, Kenneth I Berger, Roberto Giugliani, Paul Harmatz, Christoph Kampmann, William G Mackenzie, Julian Raiman, Martha Solano Villarreal, Ravi Savarirayan

Journal: Am. J. Med. Genet. A. 2015 Jan;167A(1):11-25.

 

Morquio A syndrome (mucopolysaccharidosis IVA) is a lysosomal storage disorder associated with skeletal and joint abnormalities and significant non-skeletal manifestations including respiratory disease, spinal cord compression, cardiac disease, impaired vision, hearing loss, and dental ...

Last Updated: 22 Dec 2014

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Morquio A syndrome: diagnosis and current and future therapies.
 

Author(s): Shunji Tomatsu, Eriko Yasuda, Pravin Patel, Kristen Ruhnke, Tsutomu Shimada, William G Mackenzie, Robert Mason, Mihir M Thacker, Mary Theroux, Adriana M Montaño, Carlos J Alméciga-Díaz, Luis A Barrera, Yasutsugu Chinen, William S Sly, Daniel Rowan, Yasuyuki Suzuki, Tado Orii

Journal: Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1():141-51.

 

Morquio A syndrome is an autosomal recessive disorder, one of 50 lysosomal storage diseases (LSDs), and is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Deficiency of this enzyme causes specific glycosaminoglycan (GAG) accumulation: keratan sulfate ...

Last Updated: 27 Oct 2014

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Symptoms, Diagnosis, and Treatment

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Clinical Trial Information This information is provided by ClinicalTrials.gov

Biomarker for Morquio Disease
 

Status: Recruiting

Condition Summary: Lysosomal Storage Diseases; Morquio Disease

 

Last Updated: 7 May 2015

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Gait Analysis in MPS IVA
 

Status: Recruiting

Condition Summary: MPS IVA; Morquio Syndrome

 

Last Updated: 9 Aug 2013

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Longitudinal Studies of Brain Structure and Function in MPS Disorders
 

Status: Recruiting

Condition Summary: Mucopolysaccharidosis Type I; Mucopolysaccharidosis Type II; Mucopolysaccharidosis Type VI; Mucopolysaccharidosis Type IV; Mucopolysaccharidosis Type VII

 

Last Updated: 28 Jul 2015

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