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Gaucher Disease Treatment - Forecasts from 2019 to 2024

  • ID: 4835504
  • Report
  • 101 pages
  • Knowledge Sourcing Intelligence LLP
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FEATURED COMPANIES

  • ADIENNE Pharma & Biotech
  • AVROBIO, Inc.
  • ORPHAZYME A/S
  • Pfizer Inc.
  • Sanofi Genzyme
  • MORE
The gaucher disease treatment market is estimated to be valued at US$1,673.076 million and is projected to grow at a healthy rate during the forecast period. Gaucher’s disease is a genetic disease leading to fatty substances (sphingolipids) accumulation in cells and certain organs. The disease is caused by a hereditary deficiency of the enzyme beta-glucocerebrosidase (GCase). In the case of the defective enzyme, glucosylceramide can collect in the spleen, liver, kidneys, lungs, brain, and bone marrow. The severity of Gaucher disease varies, but it is ultimately fatal. The growing demand for rare disease treatment is one of the major factors driving the growth of the global gaucher disease treatment market. Treatment for the Type 1 Gaucher is available, but for the other stages, type 2 and type 3, there is at present no cure. Technological advancements and rising R&D investments will promote the development of new drugs creating an opportunity for the growth of the gaucher disease treatment market in the forecast period and beyond. Gaucher disease is more common among Jews of Ashkenazi (Eastern European) descent, occurring in approximately 1 in 450 within this population (National Gaucher Foundation). Europe is expected to hold a significant market share owing to the disease prevalence in this region.

This research study examines the current market trends related to the demand, supply, and sales, in addition to the recent developments. Major drivers, restraints, and opportunities have been covered to provide an exhaustive picture of the market. The analysis presents in-depth information regarding the development, trends, and industry policies and regulations implemented in each of the geographical regions. Further, the overall regulatory framework of the market has been exhaustively covered to offer stakeholders a better understanding of the key factors affecting the overall market environment.

Identification of key industry players in the industry and their revenue contribution to the overall business or relevant segment aligned to the study have been covered as a part of competitive intelligence done through extensive secondary research. Various studies and data published by industry associations, analyst reports, investor presentations, press releases and journals among others have been taken into consideration while conducting the secondary research. Both bottom-up and top down approaches have been utilized to determine the market size of the overall market and key segments. The values obtained are correlated with the primary inputs of the key stakeholders in the gaucher disease treatment value chain. The last step involves complete market engineering which includes analyzing the data from different sources and existing proprietary datasets while using various data triangulation methods for market breakdown and forecasting.

Market intelligence is presented in the form of analysis, charts, and graphics to help the clients in gaining faster and efficient understanding of the gaucher disease treatment market.

Major industry players profiled as part of the report are Sanofi Genzyme, Shire Plc (Takeda Pharmaceutical Company Limited), Pfizer Inc., and ORPHAZYME A/S among others.

Segmentation

The gaucher disease treatment market has been analyzed through following segments:

By Treatment

Enzyme Replacement Therapy (ERT)
Substrate Reduction Therapy (SRT)
Other treatments for symptoms

By Geography

North America
USA
Canada
Mexico

South America
Brazil
Argentina
Others

Europe
Germany
France
United Kingdom
Spain
Others

Middle East and Africa
Saudi Arabia
Israel
Others

Asia Pacific
China
Japan
South Korea
India
Others
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • ADIENNE Pharma & Biotech
  • AVROBIO, Inc.
  • ORPHAZYME A/S
  • Pfizer Inc.
  • Sanofi Genzyme
  • MORE
1. INTRODUCTION

2. RESEARCH METHODOLOGY

3. EXECUTIVE SUMMARY

4. MARKET DYNAMICS
4.1. Market Overview and Segmentation
4.2. Market Drivers
4.3. Market Restraints
4.4. Market Opportunities
4.5. Prevalence
4.5.1. By age group
4.5.2. By gender
4.5.3. By region
4.6. Product Pipeline
4.7. Innovations

5. GAUCHER DISEASE TREATMENT MARKET BY TREATMENT
5.1. Enzyme Replacement Therapy (ERT)
5.2. Substrate Reduction Therapy (SRT)
5.3. Other treatments for symptoms

6. GAUCHER DISEASE TREATMENT MARKET BY GEOGRAPHY
6.1. North America
6.1.1. USA
6.1.2. Canada
6.1.3. Mexico
6.2. South America
6.2.1. Brazil
6.2.2. Argentina
6.2.3. Others
6.3. Europe
6.3.1. Germany
6.3.2. France
6.3.3. United Kingdom
6.3.4. Spain
6.3.5. Others
6.4. Middle East and Africa
6.4.1. Saudi Arabia
6.4.2. Israel
6.4.3. Others
6.5. Asia Pacific
6.5.1. China
6.5.2. Japan
6.5.3. South Korea
6.5.4. India
6.5.5. Others

7. COMPETITIVE INTELLIGENCE
7.1. Competitive Benchmarking and Analysis
7.2. Recent Investments and Deals
7.3. Strategies of Key Players

8. COMPANY PROFILES
8.1. Sanofi Genzyme
8.2. Shire Plc (Takeda Pharmaceutical Company Limited)
8.3. Pfizer Inc.
8.4. ORPHAZYME A/S
8.5. AVROBIO, Inc.
8.6. JCR Pharmaceutical Co., Ltd.
8.7. ADIENNE Pharma & Biotech
8.8. Actelion Pharmaceuticals UK Ltd
Note: Product cover images may vary from those shown
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  • Sanofi Genzyme
  • Shire Plc (Takeda Pharmaceutical Company Limited)
  • Pfizer Inc.
  • ORPHAZYME A/S
  • AVROBIO, Inc.
  • JCR Pharmaceutical Co., Ltd.
  • ADIENNE Pharma & Biotech
  • Actelion Pharmaceuticals UK Ltd
Note: Product cover images may vary from those shown
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The research study is based on the publisher's proprietary research model. The process involves sourcing of information from various industry experts, key professionals, decision-makers and various other stakeholders along with the validation of the responses received from the vast in-house database and premium paid databases. Publicly available sources were also taken into consideration for the final market estimates and forecast for the segments and sub-segments.

The first phase of the research process is the identification of key industry leaders, experts, decision-makers and C-level executives and then conducting interviews and surveys to gain vital insights about the market. Simultaneously, extensive secondary research is done, and key secondary data is collected as part of this process. This secondary data is collected from various databases, industry journals, whitepapers, company annual reports, research publications, and other paid data sources and relevant literature. Company profiles which include strategies, new product developments, recent investments, major products and services, financials and business plans are sourced from the annual reports, SEC filings, industry associations to preserve the quality of the information being displayed. The findings from both the primary and the secondary research process combined go under further processing for market breakdown and forecasting.

The last phase is providing intelligence in the form of presentation, charts, graphics and other different formats helping the clients in a faster and efficient understanding of the market. Under this phase complete market engineering is involved which includes analyzing the collected data from different sources and existing proprietary datasets while using various data triangulation methods for market breakdown and forecasting. The publisher has developed their market forecasting methodology that, in addition to the main market values, also takes into account other macroeconomic factors (global or local) that might have some bearing on the market under study. Taking all the necessary data, a robust forecast is developed using an algorithm that involves both static (multivariate) as well as dynamic (time-series) regression analysis. Special care is taken to make sure their algorithm passes the necessary statistical tests, and the mean prediction for future years obtained is reliable with a very high confidence level. Following data validation, the analysts begin to write the report presenting insights with the help of charts, graphs, and tables.

 

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