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Benefits for other illnesses

Other Lysosomal Storage Diseases that may benefit from the therapies mentioned:

Lysosomal Storage Diseases are a group of more than 40 different inherited disorders. These diseases are usually caused by a deficiency of a single lysosomal enzyme. Although the expression patterns differ between specific lysosomal enzymes and the clinical course may vary, virtually all of the lysosomal enzymes are modified and targeted similarly. Late Infantile Battens is a good model disorder to pilot therapy technologies on, it’s an ‘easy’ rare disease to solve given that it is truly an enzyme deficient disorder. Developing a clinical trial for Late Infantile Battens will set the stage for treatments for the other 40+ Lysosomal Storage Disorders:

Sphingolipids (number of deficiencies: 12)
Fabry, Farber, Gaucher, GM1-Gangliosidosis, Krabbe, Metachromaticleucodystrophy, Niemann-Pick types A and B, Sandhoff, Tay Sachs

Mucopolysaccharides (number of deficiencies: 10)
Hurler (MPS-I), Scheie (MPS-I), Hunter (MPS-II), Sanfilippo ((MPS-IIIA,B,C,D), Morquio (MPS-IVA,B), Maroteaux-Lamy (MPS-VI), Sly (MPS-VII)

Glycogen (number of deficiencies: 2)

Oligosaccharides (number of deficiencies: 6)
Aspartylglucosaminuria, Fucosidosis, Mannosidosis, Schindler, Sialidosis

Multiple compounds (number of deficiencies: 3)
Galactosialidosis, Mucolipidosis types II and III, Multiple sulphatase deficiency, Pseudo-Hurler dystrophy, I-Cell disease

Cholesterol (number of deficiencies: 3)
Niemann-Pick type C1 & C2, Wolman

Amino acids/sugars (number of deficiencies: 3)
Cystinosis, Infantile Sialic Acid Storage, Salla

Peptides (number of deficiencies: 1)

Proteins (number of deficiencies: 5)
Batten, Ceroid Lipofuscinosis

Lysosomal Diseases Australia: this site contains a good brief overview, including information on phenotype, enzymes, therapies and prevalence.

The Lysosomal Storage Diseases Network provides a series of pages in its site with good summary descriptions of causes, diagnosis, management, genetics and incidence. An interactive glossary enables rapid explanation of technical terms, and links are provided to other articles on these diseases.