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Gaucher disease is a genetic disorder where fat-laden Gaucher cells build up in areas like the spleen, liver and bone marrow. A person will get Gaucher Disease if both parents are carriers of the disease. In this inherited disorder where the body does not make enough glucocerebrosidase (GCase), an enzyme that breaks down fatty chemicals in the body. GCase is often mistakenly referred to as GBA, which is actually the gene that affects GCase production. Type 1: Gaucher disease type 1 makes up the majority of cases in western countries. Symptoms and signs can include bone and organ problems, but brain development is normal. This form of the disease is treatable. Find out more about type 1. Type 2: Also called acute infantile neuronopathic Gaucher disease, Gaucher disease type 2 has an onset of central nervous system problems that are typically fatal within 2 years. The central nervous system problems of type 2 are not treatable. Type 3: Gaucher disease type 3 is the most common variant of the disease worldwide. Symptoms and signs can include bone and organ problems, as well as neurological effects such as seizures. It typically has a later onset compared with type 2, and patients may survive into adulthood.
Enzyme replacement therapy (ERT) and cost:Enzyme replacement therapy (ERT) balances low levels of GCase in patients with Gaucher disease so their bodies can break down glucocerebroside. (This compensates for the missing enzyme, which is why the therapy is called enzyme replacement.) ERT involves receiving intravenous (IV) infusions about every 2 weeks, either at an infusion center or at home. In the U.S. ERT can be very expensive, totaling up to USD $200,000 or more each year. Appropriate insurance may cover most of this cost, and resources are available.
Substrate reduction therapy (SRT)Substrate reduction therapy (SRT) is a newer treatment that works differently than ERT. SRT is an oral medication that decreases the amount of glucocerebroside that the body makes, reducing excess buildup. Zavesca cost around $27000 for 90 capsules while generic Zavesca cost around $20,000 in the United States without insurance. With insurance, they are available from $50 to $26200. While Cerdelga costs upwards of $40000 for 90 capsules without insurance. Currently, there are no generics available for Cerdelga. In 2020, the global Gaucher Disease market size was XX million US$ and it is expected to reach XX million US$ by the end of 2026, with a CAGR of during 2021-2026. This report focuses on the global Gaucher Disease status, future forecast, growth opportunity, key market, and key players. The study objectives are to present the Gaucher Disease development in the United States, Europe, Asia Pacific, and the Middle East and Africa.
The key players covered in this study
- Actelion Pharma
- Takeda Pharmaceuticals America
- CoTherix, Inc.
- ANI Pharmaceuticals, Inc
- Edenbridge Pharmaceuticals LLC.
- ISU Pharm
Market segment by Type, the product can be split into
- Enzyme Replacement Therapy
- Substrate Reduction Therapy
Market segment by Application, split into
- Non-Neuronopathic Gaucher Disease
- Neuronopathic Gaucher Disease
Market segment by Regions/Countries, this report covers
- United States
- Central & South America
- Middle East Asia and Africa
The study objectives of this report are:
- To analyze global Gaucher Disease status, future forecast, growth opportunity, key market and key players.
- To strategically profile the key players and comprehensively analyze their development plan and strategies.
- To define, describe, and forecast the market by product type, market, and key regions.
Table of Contents 1 Report Overview 1.1 Study Scope 1.2 Key Market Segments 1.3 Players Covered 1.4 Market Analysis by Type 1.4.1 Global Gaucher Disease Market Size Growth Rate by Type (2014-2025) 1.4.2 Enzyme Replacement Therapy 1.4.3 Substrate Reduction Therapy 1.5 Market by Application 1.5.1 Global Gaucher Disease Market Share by Application (2014-2025) 1.5.2 Non-Neuronopathic Gaucher Disease 1.5.3 Neuronopathic Gaucher Disease 1.6 Study Objectives 1.7 Years Considered 2 Global Growth Trends 2.1 Gaucher Disease Market Size 2.2 Gaucher Disease Growth Trends by Regions 2.2.1 Gaucher Disease Market Size by Regions (2014-2025) 2.2.2 Gaucher Disease Market Share by Regions (2014-2019) 2.3 Industry Trends 2.3.1 Market Top Trends 2.3.2 Market Drivers 2.3.3 Market Opportunities 3 Market Share by Key Players 3.1 Gaucher Disease Market Size by Manufacturers 3.1.1 Global Gaucher Disease Revenue by Manufacturers (2014-2019) 3.1.2 Global Gaucher Disease Revenue Market Share by Manufacturers (2014-2019) 3.1.3 Global Gaucher Disease Market Concentration Ratio (CR5 and HHI) 3.2 Gaucher Disease Key Players Head office and Area Served 3.3 Key Players Gaucher Disease Product/Solution/Service 3.4 Date of Enter into Gaucher Disease Market 3.5 Mergers & Acquisitions, Expansion Plans 4 Breakdown Data by Type and Application 4.1 Global Gaucher Disease Market Size by Type (2014-2019) 4.2 Global Gaucher Disease Market Size by Application (2014-2019) 5 United States 5.1 United States Gaucher Disease Market Size (2014-2019) 5.2 Gaucher Disease Key Players in United States 5.3 United States Gaucher Disease Market Size by Type 5.4 United States Gaucher Disease Market Size by Application 6 Europe 6.1 Europe Gaucher Disease Market Size (2014-2019) 6.2 Gaucher Disease Key Players in Europe 6.3 Europe Gaucher Disease Market Size by Type 6.4 Europe Gaucher Disease Market Size by Application 7 China 7.1 China ... | read more...