Current and emerging management options for patients with Morquio A syndrome
Mohamed F Algahim, G Hossein AlmassiDivision of Cardiothoracic Surgery, Medical College of Wisconsin, Milwaukee, WI, USAAbstract: Morquio A syndrome is a lysosomal storage disease associated with mucopolysaccharidosis. It is caused by a deficiency of the lysosomal enzyme, N-acetylgalactosamine-6-sul...
Saved in:
| Main Authors: | , |
|---|---|
| Format: | Book |
| Published: |
Dove Medical Press,
2013-02-01T00:00:00Z.
|
| Subjects: | |
| Online Access: | Connect to this object online. |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
MARC
| LEADER | 00000 am a22000003u 4500 | ||
|---|---|---|---|
| 001 | doaj_adcd7cc2be7f4fdc9ac63c48ee8cabc0 | ||
| 042 | |a dc | ||
| 100 | 1 | 0 | |a Algahim MF |e author |
| 700 | 1 | 0 | |a Almassi GH |e author |
| 245 | 0 | 0 | |a Current and emerging management options for patients with Morquio A syndrome |
| 260 | |b Dove Medical Press, |c 2013-02-01T00:00:00Z. | ||
| 500 | |a 1176-6336 | ||
| 500 | |a 1178-203X | ||
| 520 | |a Mohamed F Algahim, G Hossein AlmassiDivision of Cardiothoracic Surgery, Medical College of Wisconsin, Milwaukee, WI, USAAbstract: 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.Keywords: lysosomal, storage disease, mucopolysaccharidosis | ||
| 546 | |a EN | ||
| 690 | |a Therapeutics. Pharmacology | ||
| 690 | |a RM1-950 | ||
| 655 | 7 | |a article |2 local | |
| 786 | 0 | |n Therapeutics and Clinical Risk Management, Vol 2013, Iss default, Pp 45-53 (2013) | |
| 787 | 0 | |n http://www.dovepress.com/current-and-emerging-management-options-for-patients-with-morquio-a-sy-a12173 | |
| 787 | 0 | |n https://doaj.org/toc/1176-6336 | |
| 787 | 0 | |n https://doaj.org/toc/1178-203X | |
| 856 | 4 | 1 | |u https://doaj.org/article/adcd7cc2be7f4fdc9ac63c48ee8cabc0 |z Connect to this object online. |