Lysosomal Storage Diseases Therapeutics’ Demand Growing With Increasing Prevalence Of Lysosomal Storage Diseases
Lysosomal storage diseases are inherited metabolic diseases caused by faulty activity of a number of enzymes in the lysosome. Patients with these diseases are likely to experience an array of symptoms, which may range from fatigue to loss of appetite. Clinical symptoms are dependent on the type of accumulated substrate, and the time it takes to develop symptoms is dependent on the disease. More than 40 types of lysosomal storage diseases have been identified by scientists. Each one affects different organs and tissues. Patients with Gaucher disease have enlargement of the spleen, problems with blood and bone development, and other symptoms that can be mild or severe. Type 1 Gaucher disease affects the central nervous system and can cause neurological symptoms. Research on the development of treatments for Lysosomal Storage Diseases Therapeutics has yielded impressive results in the last two decades. Still, there are no treatments for almost two-thirds of lysosomal diseases. D...