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

Bone mineral density in MPS IV A (Morquio syndrome type A).
 

Author(s): Heidi H Kecskemethy, Francyne Kubaski, H T Harcke, Shunji Tomatsu

Journal: Mol. Genet. Metab.. 2016 Feb;117(2):144-9.

 

Mucopolysaccharidosis IV A (MPS IV A), Morquio A, is caused by deficiency in lysosomal enzyme N-acetylgalactosamine-6-sulfate sulfatase (GALNS), which is responsible for the catabolism of the glycosaminoglycans (GAGs) keratan sulfate (KS) and chondroitin 6-sulfate (C6S). Accumulation ...

Last Updated: 15 Feb 2016

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Hematopoietic stem cell transplantation for Morquio A syndrome.
 

Author(s): Hiromasa Yabe, Akemi Tanaka, Yasutsugu Chinen, Shunichi Kato, Kazuki Sawamoto, Eriko Yasuda, Haruo Shintaku, Yasuyuki Suzuki, Tadao Orii, Shunji Tomatsu

Journal: Mol. Genet. Metab.. 2016 Feb;117(2):84-94.

 

Morquio A syndrome features systemic skeletal dysplasia. To date, there has been no curative therapy for this skeletal dysplasia. No systemic report on a long-term effect of hematopoietic stem cell transplantation (HSCT) for Morquio A has been described. We conducted HSCT for 4 cases ...

Last Updated: 15 Feb 2016

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Obstructive airway in Morquio A syndrome, the past, the present and the future.
 

Author(s): Shunji Tomatsu, Lauren W Averill, Kazuki Sawamoto, William G Mackenzie, Michael B Bober, Christian Pizarro, Christopher J Goff, Li Xie, Tadao Orii, Mary Theroux

Journal: Mol. Genet. Metab.. 2016 Feb;117(2):150-6.

 

Patients with severe tracheal obstruction in Morquio A syndrome are at risk of dying of sleep apnea and related complications. Tracheal obstruction also leads to life-threatening complications during anesthesia as a result of the difficulty in managing the upper airway due to factors ...

Last Updated: 15 Feb 2016

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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 8 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

There are currently no related results available in GeneReviews.

 
 
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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: 25 Apr 2017

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A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
 

Status: Recruiting

Condition Summary: Mucopolysaccharidosis IV Type A; Morquio A Syndrome; MPS IVA

 

Last Updated: 19 Apr 2017

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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: 6 Jan 2017

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