Familial Chylomicronia Syndrome (FDS), a life-threatening inherited blood disorder that results from an abnormality in the function of the thyroid. This condition is more common than most people think and the only way to avoid contracting this condition is to have a regular test for levels of calcium andothyroxine (T4). Other symptoms associated with FDS include bone pain, decreased growth, decreased thyroid function, osteoporosis, increased risk of cardiovascular diseases and high cholesterol.Familial Csts is usually detected through genetic testing, but in rare instances, an imaging test is required. In mild and moderate cases, genetic counselors can perform the genetic testing, but in severe conditions, patients will require a full-blown metabolic assessment.
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According to the Familial Chylomicronemia Syndrome (FCS) Foundation report, in 2017, around 30 million people suffer from rare diseases, accounted for every 1 out of 10 people in the U.S, and also 95% of rare diseases have no Food and Drug Administration (FDA) approved treatment. Familial Chylomicronemia Syndrome is a rare disorder that affect small population worldwide. According to a report by National Organiztion for Rare Disorders (NORD), in 2017, FCS is more prevalent in French community and certain communities of the Netherland, where the prevalence is as high as every 19 to 20 per one million individuals.
Severe medical condition associated with FCS are fatty liver diseases, enlarged liver, enlarged spleen, and pancreatitis, which can be life-threatening. It is characterized by inflammation of pancreas, which is very painful and can be severe. Moreover, frequent attacks of pancreatitis may lead to long-term organ failure or death. Additionally, because of the rarity of the condition, the clinical progression of this syndrome is not well understood. This is creating a lucrative opportunity for the Familial Chylomicronemia Syndrome treatment market player in near future.
Patient with FCS, may witness increase in the triglyceride level up to 20 to 30 times as compared to normal individuals, even with medication of a low-fat diet. Scientist are working to produce a therapy with a potential to reduce the level of ApoC-III in the blood. ApoC-III has proven to be a protein that regulate triglycerides level in the human body. Thus, decreasing the level of ApoC-III may help to regulate the elevated levels of triglyceride, which is a major risk factor for pancreatitis.
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Non-renewal of Glybera and advent of Volanesorsen to foster the Familial Chylomicronemia Syndrome treatment market growth
In September 2017, Akcea Therapeutics Inc., a subsidiary of Ionis Pharmaceutical Inc., announced the filing of New Drug Submission (NDS) to Health Canada for Volanesorsen with the focus to develop and commercialize drugs to treat severe cardio metabolic disease caused by lipid disorder. Volanesorsen is produced to reduce the production of ApoC-III protein production in liver, which plays an important role in the regulation of plasma TG. The Health Canada approved priority review to the drug for the treatment of Familial Chylomicronemia Syndrome in Canada, which could help stimulate the access of this medicine to the people of Canada suffering from FCS and is projected to augment the growth of Familial Chylomicronemia Syndrome treatment market in this region. Akcea Therapeutics achieved successful results in its phase 3 trails – APPROACH Study and is presently conducting the APPROACH Open Label Study to further evaluate is efficacy with extended administration of the drug in FCS patients.
Glybera, a gene therapy, is produced to restore the LPL enzyme required to clear the fat-carrying chylomicron particles from the intestine. It is a one-time gene therapy procedure, which consist of a series of 60 intramuscular injections in the legs. For instance, in October 2012, the European commission granted marketing authorization of Glybera under exceptional circumstances as a treatment for small subsets of adult patients diagnosed with familial LPL deficiency, which was confirmed by genetic testing. In April 2017, uniQure announced non-renewal of Glybera gene therapy authorization in Europe, which is scheduled to expire in October 2017. The non-renewal of Glybera’s marketing authorization in Europe is expected to affect the overall market of FCS
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Table of Contents:
- Research Objective and assumption
o Research Objectives
o Assumptions
o Abbreviations - Market Overview
o Report Description
• Market Definition and Scope
o Executive Summary
• Market Snippet By Product Type
• Market Snippet By Application
• Market Snippet By Technology
• Market Snippet By End User
• Market Snippet By Region
o Coherent Opportunity Map (COM) - Market Dynamics, Regulations, and Trends Analysis
o Market Dynamics
• Drivers
• Restraints
• Market Opportunities
o Technological Trends
o PEST Analysis
o Impact Analysis - Global Familial Chylomicronemia Syndrome Treatment Market, By Product Type, 2021 – 2028 (US$ Million)
o Introduction
• Market Share Analysis, 2016 and 2028 (%)
• Y-o-Y Growth Analysis, 2021 – 2028
• Segment Trends
o Reagents & Consumables
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
o Software’s
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
o Instruments
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million) - Global Familial Chylomicronemia Syndrome Treatment Market, By Application, 2021 – 2028 (US$ Million)
o Introduction
• Market Share Analysis, 2016 and 2028 (%)
• Y-o-Y Growth Analysis, 2021 – 2028
• Segment Trends
o Genomics
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
o Proteomics
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
o Diagnostics
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
o Drug Discovery
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million) - Global Familial Chylomicronemia Syndrome Treatment Market, By Technology, 2021 – 2028 (US$ Million)
o Introduction
• Market Share Analysis, 2016 and 2028 (%)
• Y-o-Y Growth Analysis, 2021 – 2028
• Segment Trends
o Microarrays
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
o Microfluidics
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million) - Global Familial Chylomicronemia Syndrome Treatment Market, By End User, 2021 – 2028 (US$ Million)
o Introduction
• Market Share Analysis, 2016 and 2028 (%)
• Y-o-Y Growth Analysis, 2021 – 2028
• Segment Trends
o Hospitals
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
o Diagnostic Labs
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
o Academic & Research Institutes
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
o Biotechnology & Pharmaceutical Companies
• Introduction
• Market Size and Forecast, and Y-o-Y Growth, 2021 – 2028, (US$ Million)
8. Global Familial Chylomicronemia Syndrome Treatment Market, By Regions, 2021 – 2028 (US$ Million)
o Introduction
• Market Share Analysis, By Region, 2016 and 2028 (%)
• Y-o-Y Growth Analysis, For Regions, 2021 – 2028
o North America
• Market Size and Forecast, By Product Type, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Application, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Technology, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By End User, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Country, 2021 – 2028 (US$ Million)
• U.S.
• Canada
o Europe
• Market Size and Forecast, By Product Type, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Application, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Technology, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By End User, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Country, 2021 – 2028 (US$ Million)
• Germany
• U.K.
• France
• Italy
• Spain
• Russia
• Rest of Europe
o Asia Pacific
• Market Size and Forecast, By Product Type, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Application, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Technology, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By End User, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Country, 2021 – 2028 (US$ Million)
• China
• India
• Japan
• Australia
• South Korea
• ASEAN
• Rest of APAC
o Latin America
• Market Size and Forecast, By Product Type, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Application, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Technology, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By End User, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Country, 2021 – 2028 (US$ Million)
• Brazil
• Mexico
• Rest of Latin America
o Africa
• Market Size and Forecast, By Product Type, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Application, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Technology, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By End User, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Country, 2021 – 2028 (US$ Million)
• South Africa
• Central Africa
• North Africa
o Middle East
• Market Size and Forecast, By Product Type, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Application, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Technology, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By End User, 2021 – 2028 (US$ Million)
• Market Size and Forecast, By Country, 2021 – 2028 (US$ Million)
• GCC
• Israel
• Rest of Middle East
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