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 storagedisorder 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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