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

Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study.
 

Author(s): Brian Long, Troy Tompkins, Celeste Decker, Lynne Jesaitis, Shahid Khan, Peter Slasor, Paul Harmatz, Charles A O'Neill, Becky Schweighardt

Journal: Clin Ther. 2017 Jan;39(1):118-129.e3.

 

Elosulfase alfa is an enzyme replacement therapy for the treatment of Morquio A syndrome (mucopolysaccharidosis IVA), a lysosomal storage disorder caused by a deficiency of the enzyme N-acetylgalactose-amine-6-sulfatase. We previously reported immunogenicity data from our 24-week ...

Last Updated: 13 Dec 2016

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Antibodies that neutralize cellular uptake of elosulfase alfa are not associated with reduced efficacy or pharmacodynamic effect in individuals with Morquio A syndrome.
 

Author(s): Andrew C Melton, Russell K Soon, Troy Tompkins, Brian Long, Becky Schweighardt, Yulan Qi, Catherine Vitelli, Anil Bagri, Celeste Decker, Charles A O'Neill, Stephen J Zoog, Lynne Jesaitis

Journal: J. Immunol. Methods. 2017 Jan;440():41-51.

 

Many enzyme replacement therapies (ERTs) for lysosomal storage disorders use the cell-surface cation-independent mannose-6 phosphate receptor (CI-M6PR) to deliver ERTs to the lysosome. However, neutralizing antibodies (NAb) may interfere with this process. We previously reported that ...

Last Updated: 28 Oct 2016

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Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study.
 

Author(s): Paul R Harmatz, Eugen Mengel, Tarekegn Geberhiwot, Nicole Muschol, Christian J Hendriksz, Barbara K Burton, Elisabeth Jameson, Kenneth I Berger, Andrea Jester, Marsha Treadwell, Zlatko Sisic, Celeste Decker

Journal: Am. J. Med. Genet. A. 2017 Feb;173(2):375-383.

 

Efficacy and safety of elosulfase alfa enzyme replacement therapy (ERT) were assessed in an open-label, phase 2, multi-national study in Morquio A patients aged ≥5 years unable to walk ≥30 meters in the 6-min walk test. Patients received elosulfase alfa 2.0 mg/kg/week intravenously ...

Last Updated: 24 Oct 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

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Clinical Trial Information This information is provided by ClinicalTrials.gov

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
 

Status: Recruiting

Condition Summary: Morquio Disease; MPS - Mucopolysaccharidosis

 

Last Updated: 10 May 2017

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Biomarker for Morquio Disease
 

Status: Recruiting

Condition Summary: Lysosomal Storage Diseases; Morquio Disease

 

Last Updated: 25 Apr 2017

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Natural History of Atypical Morquio A Disease
 

Status: Not yet recruiting

Condition Summary: Mucopolysaccharidosis IV A

 

Last Updated: 29 Jun 2017

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