Title |
Current and emerging management options for patients with Morquio A syndrome
|
---|---|
Published in |
Therapeutics and Clinical Risk Management, February 2013
|
DOI | 10.2147/tcrm.s24771 |
Pubmed ID | |
Authors |
G. Hossein Almassi, Algahim |
Abstract |
Morquio A syndrome is a lysosomal storage disease associated with mucopolysaccharidosis. It is caused by a deficiency of the lysosomal enzyme, N-acetylgalactosamine-6-sulfate sulfatase, which leads to accumulation of keratan sulfate and condroitin-6 sulfate in multiple organs. Patients present with multisystemic complications involving the musculoskeletal, respiratory, cardiovascular, and digestive systems. Presently, there is no definitive cure, and current management options are palliative. Enzyme replacement therapy and hematopoietic stem cell therapy have been proven effective in certain lysosomal storage diseases, and current investigations are underway to evaluate the effectiveness of these therapies and others for the treatment of Morquio A syndrome. This review discusses the current and emerging treatment options for Morquio A syndrome, citing examples of the treatment of other mucopolysaccharidoses. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
Australia | 1 | 33% |
Unknown | 2 | 67% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 3 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
United Kingdom | 1 | 2% |
Spain | 1 | 2% |
Unknown | 52 | 96% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Master | 9 | 17% |
Student > Bachelor | 9 | 17% |
Researcher | 7 | 13% |
Student > Ph. D. Student | 4 | 7% |
Student > Doctoral Student | 3 | 6% |
Other | 7 | 13% |
Unknown | 15 | 28% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 20 | 37% |
Neuroscience | 4 | 7% |
Nursing and Health Professions | 3 | 6% |
Agricultural and Biological Sciences | 3 | 6% |
Chemistry | 2 | 4% |
Other | 6 | 11% |
Unknown | 16 | 30% |