Sphingolipidoses Treatment Market Segmented By indication in Fabry Disease, Batten Disease, Gaucher Disease, NiemannPick Disease, Krabbe Disease, Tay-Sach Disease with therapy type such as Enzyme Replacement Therapy, Stem Cell Therapy, Substrate Reduction Therapy, Chaperone Therapy
Industry: Healthcare
Format: PPT*, PDF, EXCEL
Delivery Timelines: Contact Sales
Report Type: Ongoing
Report ID: PMRREP31575
The sphingolipidoses are a group of inherited metabolic disorder caused due to deficiency of specific enzymes in the body. It is also known as lysosomal storage disorder. Lysosome is present inside the cell and is responsible for breakdown of cell substances that speeded up the reaction in the body.
Lysosomal disorders occur due to absence of lysosomal enzymes in the body which results in development of toxic substance and thereby contributing in damage of organs. The disorder primarily hit the sites of sphingolipid catabolism like lysosomes of phagocytes, histiocytes or macrophages in the bone marrow, spleen and liver get affected. Sphingolipidoses is usually more prevalent amongst pediatric population.
The degenerative pattern of disease is characterized by involvement of diffuse and progressive grey matter with psychomotor retardation. A cherry-red at the macula and optic atrophy are the common sign during the diagnosis of this disease.
The disease is autosomal recessive in nature that results in enlarge liver and spleen and mental retardation. Specific lysosomal enzyme defects includes Niemann-Pick disease, Krabbe’s disease, Gaucher’s disease, Glycogen Storage disease II, Tay-Sachs disease and some other.
All are rare metabolic disorders. Symptoms of sphingolipidoses includes fever, tingling, numbness, pain or burning in the feet and hand, tiredness, skin sores, dizziness and so on.
The sphingolipidoses treatment market is expected to witness a rapid growth during the forecast period due to factor such as allocation of twenty-three orphan drugs approved by FDA used for sphingolipidoses treatment.
Moreover, rising incidence of lysosomal disease, increase in diagnostic rate, awareness about the rare disorders will create a demand to go for therapeutic treatment which will ultimately impact the market positively during the forecast period.
Additionally, increase focus on R&D for diagnosis and drug development for sphingolipidoses treatment and implementation of orphan drugs that provide marketing and profitable benefit to pharmaceutical companies will also be contributing to drive the growth of sphingolipidoses treatment market.
However, high cost of the available treatment, heterogeneity of the disease leading to misdiagnosis of sphingolipidoses and lack of diverse treatment option will hamper the market growth for sphingolipidoses treatment.
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Some of the key players for sphingolipidoses treatment market operating at the global level include Shire, Sanofi, Pfizer, Inc., Actelion Pharmaceuticals Ltd., BioMarin, Merck & Co., Inc., Raptor Pharmaceutical Corp., BioMarin Pharmaceutical Inc., Protalix Biotherapeutics Inc., Amicus Therapeutics, Inc., Novartis AG, Teva Pharmaceutical Industries Ltd. and others
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