About Lysosomal Storage Diseases
Lysosomal storage disorders are a set of hereditary metabolic diseases that are caused by enzyme deficits in different bodily cells. As a result of enzyme shortages, lysosomal storage disorders are inherited metabolic illnesses defined by an abnormal build-up of numerous harmful chemicals in the body's cells. There are approximately 50 of these ailments in all, and they can affect the skeleton, brain, skin, heart, and central nervous system, among other bodily components. New lysosomal storage diseases are always being discovered. While several of these disorders are undergoing clinical trials, there is presently no approved medication for many lysosomal storage diseases.The key factor driving the growth of the worldwide lysosomal storage disorders therapeutics market is an increase in diagnosis rates due to increased awareness and financial incentives for orphan drug development to recover R&D costs. Other reasons driving global demand for lysosomal storage disorders therapies include increased attention of pharmaceutical companies on research and development of medications for the treatment of uncommon diseases, as well as speedier uptake and market access.
Attributes | Details |
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Study Period | 2018-2030 |
Base Year | 2023 |
Unit | Value (USD Million) |
The market for lysosomal storage disorder treatment is very competitive, with only a few large competitors. Few big firms currently dominate the market in terms of market share. The major players are doing extensive research to develop new medication and a proper treatment for the disease. Analyst at AMA Research estimates that United States Players will contribute the maximum growth to Global Lysosomal Storage Diseases market throughout the forecasted period. Established and emerging Players should take a closer view at their existing organizations and reinvent traditional business and operating models to adapt to the future.
Shire PLC (United States), Pfizer, Inc (United States), Sanofi (France), BioMarin Pharmaceutical Inc. (United States), Actelion Ltd. (Switzerland), Raptor Pharmaceutical Corp.(United States), Protalix Biotherapeutics Inc. (Israel), Amicus Therapeutics, Inc.(United States), Orphazyme A/S (Denmark) and GC Pharma (South Korea) are some of the key players that are part of study coverage. Additionally, the Players which are also part of the research coverage are Cyclo Therapeutics (United States) and CANbridge Life Sciences Ltd. (China).
Segmentation Overview
AMA Research has segmented the market of Global Lysosomal Storage Diseases market by , Application (Hospitals and Ambulatory Surgical Centers) and Region.
On the basis of geography, the market of Lysosomal Storage Diseases has been segmented into South America (Brazil, Argentina, Rest of South America), Asia Pacific (China, Japan, India, South Korea, Taiwan, Australia, Rest of Asia-Pacific), Europe (Germany, France, Italy, United Kingdom, Netherlands, Rest of Europe), MEA (Middle East, Africa), North America (United States, Canada, Mexico). If we see Market by Treatment, the sub-segment i.e. Enzyme Replacement Therapy will boost the Lysosomal Storage Diseases market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth. If we see Market by Indication, the sub-segment i.e. Gaucher disease will boost the Lysosomal Storage Diseases market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth.
Influencing Trend:
Enzyme Replacement Therapy
Market Growth Drivers:
Increasing Incidences of Lysosomal Diseases and Growing Awareness and Increasing Diagnosis Rate
Challenges:
Lack of Treatment Options and Stringent Regulatory Factors
Restraints:
High Cost of Treatment and Underdiagnoses of Lysosomal Storage Diseases
Opportunities:
Increased Investment for Drug Development and Increasing Focus of Pharmaceutical Companies in The Research and Development of Drugs for the Treatment of Rare Diseases
Market Leaders and their expansionary development strategies
In April 2023, Centogene a provider of data-driven answers in rare and neurodegenerative diseases, has extended its partnership with Takeda to diagnose patients with lysosomal storage disorders. Under the renewed one-year partnership agreement, Centogene says it will continue to provide Takeda with access to diagnostic testing for patients around the world.
In February 2020, Takeda to Present Research Advances in Rare Lysosomal Storage. Takeda is also supporting, through an educational grant, a satellite symposium on building a better roadmap for the care of Gaucher patients and sponsoring a satellite symposium on peripheral, central and autonomic neurodegenerative consequences in Lysosomal Disease
Key Target Audience
Drug Companies, Pharmaceutical companies, Research Companies, Medical Research Companies and Others
About Approach
To evaluate and validate the market size various sources including primary and secondary analysis is utilized. AMA Research follows regulatory standards such as NAICS/SIC/ICB/TRCB, to have a better understanding of the market. The market study is conducted on basis of more than 200 companies dealing in the market regional as well as global areas with the purpose to understand the companies positioning regarding the market value, volume, and their market share for regional as well as global.
Further to bring relevance specific to any niche market we set and apply a number of criteria like Geographic Footprints, Regional Segments of Revenue, Operational Centres, etc. The next step is to finalize a team (In-House + Data Agencies) who then starts collecting C & D level executives and profiles, Industry experts, Opinion leaders, etc., and work towards appointment generation.
The primary research is performed by taking the interviews of executives of various companies dealing in the market as well as using the survey reports, research institute, and latest research reports. Meanwhile, the analyst team keeps preparing a set of questionnaires, and after getting the appointee list; the target audience is then tapped and segregated with various mediums and channels that are feasible for making connections that including email communication, telephonic, skype, LinkedIn Group & InMail, Community Forums, Community Forums, open Survey, SurveyMonkey, etc.