About Refsum Disease Treatment
Refsum disease is a metabolic disorder that causes vision loss, absence of a sense of smell, and a number of other symptoms caused by a number of faulty genes (principally PHYH). The patient suffering from this unable to degrade phytanic acid because of deficient activity of phytanoyl-CoA hydroxylase (PhyH), a peroxisomal enzyme catalyzing the first step of phytanic acid alpha-oxidation. Currently, there two treatments are available for Refsum disease including a strict diet low in phytanic acid and is used for long-term management direct removal from the bloodstream of phytanic acid by a dialysis-type process (plasmapheresis or lipid apheresis).
Attributes | Details |
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Study Period | 2018-2030 |
Base Year | 2023 |
Unit | Value (USD Million) |
The companies are exploring the market by adopting expansions, investments, new service launches and collaborations as their preferred strategies. The players are exploring new geographies through expansions and acquisitions across the globe to gain a competitive advantage through combined collaborations. Analyst at AMA Research estimates that United States Players will contribute the maximum growth to Global Refsum Disease Treatment 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.
Fresenius Kabi (Germany), B. Braun Medical Inc. (United States), Medline Industries, Inc. (United States), Illumina, Inc. (United States), Igenomix, S.L. (Spain), Ceuta Healthcare Limited (United Kingdom), Cochlear Ltd. (Australia) and Sonova (Switzerland) are some of the key players that are part of study coverage. Additionally, the Players which are also part of the research coverage are William Demant Holding A/S (Denmark), Nurotron Biotechnology Co. Ltd. (United States), Agilent Technologies (United States), Bio-Rad Laboratories, Inc. (United States) and Sequenom (United States).
Segmentation Overview
AMA Research has segmented the market of Global Refsum Disease Treatment market by Type (Surgery Therapy and Medication), Application (Hospitals, Clinics and Others) and Region.
On the basis of geography, the market of Refsum Disease Treatment 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 Diagnosis, the sub-segment i.e. Physical will boost the Refsum Disease Treatment market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth.
Influencing Trend:
Increasing Government Funding and Improvement in the Regulatory Framework
Market Growth Drivers:
Increasing Prevalence of Refsum Disease and Rise in the Awareness about Refsum Disease Treatment Options
Challenges:
Lack of Healthcare Infrasture in Underdeveloped Countries
Restraints:
Lack of Awareness in Underdevelopment Countries
Opportunities:
Growth in the Healthcare Industry Worldwide and Increasing Research and Development Activities
Market Leaders and their expansionary development strategies
In August 2023, Alexion Pharmaceuticals teams up with patient advocacy groups to develop educational resources and support programs for individuals living with Refsum disease.
In September 2023, Alexion Pharmaceuticals launches a patient support program for VITAL, providing financial assistance and access to medication for eligible patients.
Key Target Audience
Refsum Disease Treatment Providers, Emerging Companies, Research Professionals and End-users
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.