Lysosomal Storage Disorder Therapeutics – Analysis, Clinical Trials & Results, 2017

Lysosomal storage disorder refers to inherited metabolic disorders that are characterize by enzyme deficiencies, which results in an abnormal build-up of various toxic materials in the body’s cells. There are nearly 50 types of lysosomal storage disorder which affect various parts of the body including the brain, skeleton, heart, central nervous system and skin.

Some of the lysosomal storage disorders are Aspartylglucosaminuria, Batten Disease, Cystinosis, Fabry Disease, Glycogen Storage Disease II (Pompe Disease), GM2-Gangliosidosis Type I (Tay Sachs Disease), GM2-Gangliosidosis Type II (Sandhoff Disease), Metachromatic Leukodystrophy, Gaucher’s Disease Types I, II, and III, Mucolipidosis Types I, II/III and IV, Mucopolysaccharide Storage Diseases (Hurler Disease and variants, Hunter, Sanfilippo Types A,B,C,D, Morquio Types A and B, Maroteaux-Lamy and Sly diseases), Niemann-Pick Disease Types A/B, C1 and C2 and Schindler Disease Types I and II.

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Drug manufacturing companies are constantly involved in the quest to find better and new treatment strategies with potential enzyme replacement therapies such as N-acetylglucosaminidase alpha (NAGLU) replacement which can also prove to be effective in Sanfilippo syndrome type B treatment. Various companies are developing drug candidates using different technologies for the development of effective and better drugs, which is driving the growth of the pipeline.

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Some of the other key players developing drugs for the treatment of lysosome storage disorder include GlaxoSmithKline plc, Sanofi Genzyme, Amicus Therapeutics, Inc. and others.


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