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 34 free, full-text research articles on human participants. First 3 results:

Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y.
 

Author(s): Simon A Jones, Martin Bialer, Rossella Parini, Ken Martin, Hui Wang, Ke Yang, Adam J Shaywitz, Paul Harmatz

Journal: Pediatr. Res.. 2015 Dec;78(6):717-22.

 

Previous studies have shown that elosulfase alfa has a favorable efficacy/safety profile in Morquio A patients aged ≥5 y. This study evaluated safety and impact on urine keratan sulfate (uKS) levels and growth velocity in younger patients.

Last Updated: 23 Dec 2015

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Diagnosis of Morquio Syndrome in Dried Blood Spots Based on a New MRM-MS Assay.
 

Author(s): Claudia Cozma, Sabrina Eichler, Gyula Wittmann, Alba Flores Bonet, Guido Johannes Kramp, Anne-Katrin Giese, Arndt Rolfs

Journal:

 

Mucopolysaccharidosis IVA (MPS IVA; Morquio A disease) is an autosomal recessive disease caused and characterized by a decreased activity of N-acetylgalactosamine-6-sulfate sulfatase (GALNS), resulting in accumulation of keratan sulfate and chondroitin-6-sulfate in tissues and secondary ...

Last Updated: 7 Jul 2015

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Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study.
 

Author(s): Barbara K Burton, Kenneth I Berger, Gregory D Lewis, Mark Tarnopolsky, Marsha Treadwell, John J Mitchell, Nicole Muschol, Simon A Jones, V Reid Sutton, Gregory M Pastores, Heather Lau, Rebecca Sparkes, Fred Genter, Adam J Shaywitz, Paul Harmatz

Journal: Am. J. Med. Genet. A. 2015 Oct;167A(10):2272-81.

 

The primary treatment outcomes of a phase 2, randomized, double-blind, pilot study evaluating safety, physiological, and pharmacological effects of elosulfase alfa in patients with Morquio A syndrome are herewith presented. Patients aged ≥7 years and able to walk ≥200 m in the ...

Last Updated: 11 Sep 2015

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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 7 free, full-text review articles on human participants. First 3 results:

Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.
 

Author(s): Shunji Tomatsu, Kazuki Sawamoto, Carlos J Alméciga-Díaz, Tsutomu Shimada, Michael B Bober, Yasutsugu Chinen, Hiromasa Yabe, Adriana M Montaño, Roberto Giugliani, Francyne Kubaski, Eriko Yasuda, Alexander Rodríguez-López, Angela J Espejo-Mojica, Oscar F Sánchez, Robert W Mason, Luis A Barrera, William G Mackenzie, Tadao Orii

Journal:

 

Patients with mucopolysaccharidosis IVA (MPS IVA) can present with systemic skeletal dysplasia, leading to a need for multiple orthopedic surgical procedures, and often become wheelchair bound in their teenage years. Studies on patients with MPS IVA treated by enzyme replacement therapy ...

Last Updated: 21 Apr 2015

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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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A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases.
 

Author(s): Regina M Leadley, Shona Lang, Kate Misso, Trudy Bekkering, Janine Ross, Takeyuki Akiyama, Michael Fietz, Roberto Giugliani, Chris J Hendriksz, Ngu Lock Hock, Jim McGill, Andrew Olaye, Mohit Jain, Jos Kleijnen

Journal:

 

Morquio A (MPS IVA) is a rare disease characterised by a deficiency of N-acetylgalactosamine-6 sulfatase (GALNS) and presenting with short stature, abnormal gait, cervical spine instability and shortened lifespan.

Last Updated: 28 Apr 2015

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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: 18 May 2016

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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: 9 Mar 2016

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