A new market study, titled “Lysosomal Storage Disorder Pipeline Analysis – 2018”, has been featured on WiseGuyReports.
Th report Lysosomal Storage Disorder Pipeline Analysis – 2018 covers as detailed description of pipeline products that are in various stages of development. The key contents include the critical clinical end points considered, mechanism of action, expected end of clinical trials, entry into the market and the key companies that are working in the pipeline.
Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. There are nearly 50 of these disorders altogether, and they may affect different parts of the body, including the skeleton, brain, skin, heart, and central nervous system. New lysosomal storage disorders continue to be identified. While clinical trials are in progress on possible treatments for some of these diseases, there is currently no approved treatment for many lysosomal storage diseases.
Some of the most common lysosomal storage disorders include:
– Gaucher disease: Gaucher disease often causes spleen and liver enlargement, blood problems and bone issues. Learn more about Gaucher disease.
– Fabry disease: This disorder often causes severe burning pains in hands and feet and, in some cases, a distinctive skin rash on the legs. Untreated, this disease can cause kidney failure, heart failure, strokes and death before age 50. Although men are more likely to have severe disease, women may also be seriously affected.
– Niemann-Pick disease: Similar to Gaucher disease, Niemann-Pick disease involves organ enlargement, lung dysfunction and central nervous system damage for certain subtypes.
– Hunter syndrome: This disease is part of a group of disorders that cause bone and joint deformity as well as interference with normal growth.
– Glycogen storage disease II (Pompe disease): Depending on the specific subtype, Pompe disease may cause heart enlargement and heart failure in infants. It may also cause respiratory problems and severe muscle weakness in adults.
– Tay-Sachs disease: This disorder causes severe and fatal mental and physical deterioration, with both an early-onset and a late-onset form.
Axcentua Pharmaceuticals AB
Belrose Pharma, Inc
Fate Therapeutics, Inc.
JCR Pharmaceuticals Co., Ltd.
Lixte Biotechnology Holdings, Inc
Table of Content
- Lysosomal Storage Disorder
- Market Analysis
- Therapeutics under Development by Companies
- Last Stage Products (Phase III)
- Mid Stage Products (Phase II)
- Early Stage Products (Phase I)
- Discovery and Pre-clinical stage Products
- Therapeutic Assessment
- Discontinued Products
- Competitors Threat Assessment
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