The global lysosomal storage disease market is anticipated to grow at a significant rate during the forecast period. Lysosomal storage disease comes under rare diseases. The number of incidences for the disease is around 1 in 7,700 according to the Gaucher’s Disease Foundation. The disease has several symptoms thus it is difficult to identify and diagnose. There are more than 50 types of lysosomal storage disease which are caused by the lack of enzymes. The different types of lysosomal storage diseases include Gaucher’s disease, Fabry disease, Niemann-pick disease, hunter syndrome, Tay-Sachs disease and more. These diseases affect different body parts such as Gaucher’s disease affect the liver and cause blood and bone problems. Tay-Sachs disease cause fatal mental and physical distortions.
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The symptoms of the disease can vary from the type of lysosomal storage disease. The general symptoms are observed in the bones and joints, central nervous system, eyes, heart, kidney and lungs. The variation of the symptoms causes difficulty in the diagnosis of the disease. The diagnosis may take years for the physician to confirm the lysosomal storage disease, which is diagnosed on the basis of the pattern of symptoms observed. Gaucher’s disease can be diagnosed much easily by a simple blood test at the nearest local hospital.
The efforts are put in for the research and development to diagnose the lysosomal storage disease. The specialized testing laboratories have been made in which the disease could be diagnosed. The lysosomal storage disease treatment does not have any cure for lysosomal storage disease. The researchers are putting in constant efforts to find a solution that would address the underlying causes rather than just treating the disease.
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The treatment of disease options isvery expensive. These options include bone marrow transplantation, substrate reduction therapy, and enzyme replacement therapy. The complications and technology required for these treatments make the treatment of the disease expensive. Researchers are trying to find more affordable treatment for all kinds of lysosomal storage diseases. The constant development and research put together for the diagnosis and treatment of the disease would result in the growth of the lysosomal storage disease market.
Global Lysosomal Storage Disease Market
By Type
- Gaucher Disease
- Pompe Disease
- Tay-Sachs Disease
- Fabry Disease
- Other (Cystinosis and Krabbe Disease)
By Treatment
- Enzyme Replacement Therapy
- Substrate Reduction Therapy
- Stem Cell Transplant
Lysosomal Storage Disease Market– Segment by Region
North America
- United States
- Canada
Europe
- Germany
- United Kingdom
- France
- Spain
- Italy
- Rest of Europe
Asia-Pacific
- China
- Japan
- India
- Rest of Asia-Pacific
Rest of the World
- Middle East & Africa
- Latin America
Company Profiles
- Actelion Pharmaceuticals, Ltd. (A Part of Johnson & Johnson Service, Inc.)
- Alexion Pharmaceuticals, Inc.
- Amicus Therapeutics, Inc.
- AMO Pharma, Ltd.
- Arena Pharmaceuticals, Inc.
- BioMarin Pharmaceuticals, Inc.
- Chiesi Farmaceutici SpA
- Eli Lilly Co.
- Horizon Therapeutics, Inc.
- Leadiant Biosciences, Inc.
- Pfizer, Inc.
- Protalix Biotherapeutics, Inc.
- Sanofi S.A.
- Sigilon Therapeutics, Inc.
- Takeda Pharmaceuticals Co. Ltd.
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