Abstract

ISSN

2249-7633

e ISSN

2249-7625

Publisher

Pharmacy Practice & Drug Research

MUCOPOLYSACCHARIDOSIS TYPE IV- A (Morquio A Syndrome): A BRIEF REVIEW
Author / Afflication
CH. Chandrasekhar

Department of Pharmaceutics,Vikas Institute of Pharmaceutical Sciences, Rjahmyndry-533102, Andhra Pradesh, India
P. Achutha Lakshmi

Department of Pharmacy Practice, Vikas Institute of Pharmaceutical Sciences, Rjahmyndry-533102, Andhra Pradesh, India.
B.V.B. Balaji

Department of Pharmacy Practice, Vikas Institute of Pharmaceutical Sciences, Rjahmyndry-533102, Andhra Pradesh, India.
P. Niharika

Department of Pharmacy Practice, Vikas Institute of Pharmaceutical Sciences, Rjahmyndry-533102, Andhra Pradesh, India.
V.S.L. Deepthi

Department of Pharmacy Practice, Vikas Institute of Pharmaceutical Sciences, Rjahmyndry-533102, Andhra Pradesh, India.
M. Priya

Department of Pharmacy Practice, Vikas Institute of Pharmaceutical Sciences, Rjahmyndry-533102, Andhra Pradesh, India.
Keywords
Lysosomal ,Storage disease ,Mucopolysaccharidosis ,
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 mucopoly

Volume / Issue / Year

5 , 3 , 2015

Starting Page No / Endling Page No

121 - 127