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Glycogen Storage Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034

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    Report

  • 131 Pages
  • May 2024
  • Region: Global
  • IMARC Group
  • ID: 5970028
The 7 major glycogen storage disease markets are expected to exhibit a CAGR of 5.76% during 2023-2034.

The glycogen storage disease market has been comprehensively analyzed in this report titled "Glycogen Storage Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Glycogen storage disease, also known as glycogenosis, refers to a group of inherited metabolic disorders that affect the body's ability to process and store glycogen properly. As a result, this condition leads to an abnormal accumulation or breakdown of glycogen in various tissues. The common symptoms of the ailment may include an enlarged liver and spleen, low blood sugar, difficulty breathing, muscle weakness, growth delays or short stature, abnormal levels of fats and proteins in the blood, obesity, liver dysfunction or cirrhosis, mouth sores, heart muscle disease, etc. The diagnosis of this illness typically involves a combination of clinical examination, blood workups to evaluate enzyme activity as well as glycogen levels, and genetic testing. The healthcare provider might also perform imaging studies, such as abdominal ultrasound, to assess the pancreas and liver for signs of inflammation, structural abnormalities, or cystic lesions that may be related to the condition. Additionally, a tissue biopsy, along with histochemical staining or electron microscopy, may be necessary to confirm the diagnosis by measuring the levels of glycogen or enzyme present.

The increasing cases of genetic mutations that affect the regulation of specific enzymes, thereby leading to imbalances in glycogen synthesis or breakdown, are primarily driving the glycogen storage disease market. In addition to this, the widespread adoption of enzyme replacement therapy, which involves administering a synthetic form of the deficient enzyme intravenously or orally, is also propelling the market growth. Furthermore, the inflating utilization of xanthine oxidase inhibitors, including allopurinol and oxypurinol, for reducing the levels of uric acid in the blood and preventing the risk of complications, like gout and kidney problems, is acting as another significant growth-inducing factor. Apart from this, the escalating application of molecular diagnostic techniques, such as direct sequencing, polymerase chain reaction-based assays, hybridization, etc., to provide early detection of the illness, which allows for timely intervention and management, is also creating a positive outlook for the market. Moreover, the emerging popularity of gene therapy for treating the ailment, since it aims to correct the faulty genetic material in patients while restoring normal enzyme function and improving metabolic control, is expected to drive the glycogen storage disease market in the coming years.

This report provides an exhaustive analysis of the glycogen storage disease market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for glycogen storage disease and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the glycogen storage disease market in any manner.

Time Period of the Study

  • Base Year: 2023
  • Historical Period: 2018-2023
  • Market Forecast: 2024-2034

Countries Covered

  • United States
  • Germany
  • France
  • United Kingdom
  • Italy
  • Spain
  • Japan

Analysis Covered Across Each Country

  • Historical, current, and future epidemiology scenario
  • Historical, current, and future performance of the glycogen storage disease market
  • Historical, current, and future performance of various therapeutic categories in the market
  • Sales of various drugs across the glycogen storage disease market

Competitive Landscape:

This report also provides a detailed analysis of the current glycogen storage disease marketed drugs and late-stage pipeline drugs.

In-Market Drugs

  • Drug overview
  • Mechanism of action
  • Regulatory status
  • Clinical trial results
  • Drug uptake and market performance

Late-Stage Pipeline Drugs

  • Drug overview
  • Mechanism of action
  • Regulatory status
  • Clinical trial results
  • Drug uptake and market performance
*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

  • How has the glycogen storage disease market performed so far and how will it perform in the coming years?
  • What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
  • What was the country-wise size of the glycogen storage disease market across the seven major markets in 2023 and what will it look like in 2034?
  • What is the growth rate of the glycogen storage disease market across the seven major markets and what will be the expected growth over the next ten years?
  • What are the key unmet needs in the market?

Epidemiology Insights

  • What is the number of prevalent cases (2018-2034) of glycogen storage disease across the seven major markets?
  • What is the number of prevalent cases (2018-2034) of glycogen storage disease by age across the seven major markets?
  • What is the number of prevalent cases (2018-2034) of glycogen storage disease by gender across the seven major markets?
  • How many patients are diagnosed (2018-2034) with glycogen storage disease across the seven major markets?
  • What is the size of the glycogen storage disease patient pool (2018-2023) across the seven major markets?
  • What would be the forecasted patient pool (2024-2034) across the seven major markets?
  • What are the key factors driving the epidemiological trend of glycogen storage disease?
  • What will be the growth rate of patients across the seven major markets?

Glycogen Storage Disease: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

  • What are the current marketed drugs and what are their market performance?
  • What are the key pipeline drugs and how are they expected to perform in the coming years?
  • How safe are the current marketed drugs and what are their efficacies?
  • How safe are the late-stage pipeline drugs and what are their efficacies?
  • What are the current treatment guidelines for glycogen storage disease drugs across the seven major markets?
  • Who are the key companies in the market and what are their market shares?
  • What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the glycogen storage disease market?
  • What are the key regulatory events related to the glycogen storage disease market?
  • What is the structure of clinical trial landscape by status related to the glycogen storage disease market?
  • What is the structure of clinical trial landscape by phase related to the glycogen storage disease market?
  • What is the structure of clinical trial landscape by route of administration related to the glycogen storage disease market?
This product will be updated with the latest data at the time of order. Consequently, dispatch time for this product will be 7-10 business days.

Table of Contents

?1 Preface
2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology
3 Executive Summary
4 Glycogen Storage Disease - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence
5 Glycogen Storage Disease - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment
6 Patient Journey
7 Glycogen Storage Disease - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (2018-2034)
7.2.4 Epidemiology by Gender (2018-2034)
7.2.5 Diagnosed Cases (2018-2034)
7.2.6 Patient Pool/Treated Cases (2018-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (2018-2034)
7.3.4 Epidemiology by Gender (2018-2034)
7.3.5 Diagnosed Cases (2018-2034)
7.3.6 Patient Pool/Treated Cases (2018-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario 2018-2023
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (2018-2034)
7.4.4 Epidemiology by Gender (2018-2034)
7.4.5 Diagnosed Cases (2018-2034)
7.4.6 Patient Pool/Treated Cases (2018-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (2018-2034)
7.5.4 Epidemiology by Gender (2018-2034)
7.5.5 Diagnosed Cases (2018-2034)
7.5.6 Patient Pool/Treated Cases (2018-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (2018-2034)
7.6.4 Epidemiology by Gender (2018-2034)
7.6.5 Diagnosed Cases (2018-2034)
7.6.6 Patient Pool/Treated Cases (2018-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (2018-2034)
7.7.4 Epidemiology by Gender (2018-2034)
7.7.5 Diagnosed Cases (2018-2034)
7.7.6 Patient Pool/Treated Cases (2018-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (2018-2034)
7.8.4 Epidemiology by Gender (2018-2034)
7.8.5 Diagnosed Cases (2018-2034)
7.8.6 Patient Pool/Treated Cases (2018-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (2018-2034)
7.9.4 Epidemiology by Gender (2018-2034)
7.9.5 Diagnosed Cases (2018-2034)
7.9.6 Patient Pool/Treated Cases (2018-2034)
8 Glycogen Storage Disease - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 Glycogen Storage Disease - Unmet Needs10 Glycogen Storage Disease - Key Endpoints of Treatment
11 Glycogen Storage Disease - Marketed Products
11.1 List of Glycogen Storage Disease Marketed Drugs Across the Top 7 Markets
11.1.1 Lumizyme (Alglucosidase alfa) - Sanofi
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets
11.1.2 Nexviazyme (Avalglucosidase alfa) - Sanofi
11.1.2.1 Drug Overview
11.1.2.2 Mechanism of Action
11.1.2.3 Regulatory Status
11.1.2.4 Clinical Trial Results
11.1.2.5 Sales Across Major Markets
11.1.3 Cipaglucosidase alfa - Amicus Therapeutics
11.1.3.1 Drug Overview
11.1.3.2 Mechanism of Action
11.1.3.3 Regulatory Status
11.1.3.4 Clinical Trial Results
11.1.3.5 Sales Across Major Markets
Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.
12 Glycogen Storage Disease - Pipeline Drugs
12.1 List of Glycogen Storage Disease Pipeline Drugs Across the Top 7 Markets
12.1.1 mRNA 3745 - Moderna Therapeutics
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 DTX 401 - Ultragenyx Pharmaceutical
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status
12.1.3 Triheptanoin - Ultragenyx Pharmaceutical
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status
12.1.4 AT845 - Astellas Gene Therapies
12.1.4.1 Drug Overview
12.1.4.2 Mechanism of Action
12.1.4.3 Clinical Trial Results
12.1.4.4 Safety and Efficacy
12.1.4.5 Regulatory Status
Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.
13. Glycogen Storage Disease - Attribute Analysis of Key Marketed and Pipeline Drugs
14. Glycogen Storage Disease - Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events
15. Glycogen Storage Disease - Market Scenario15.1 Market Scenario - Key Insights
15.1 Market Scenario - Top 7 Markets
15.1.1 Glycogen Storage Disease - Market Size
15.1..1.1 Market Size (2018-2023)
15.1.1.2 Market Forecast (2024-2034)
15.1.2 Glycogen Storage Disease - Market Size by Therapies
15.1.2.1 Market Size by Therapies (2018-2023)
15.1.2.2 Market Forecast by Therapies (2024-2034)
15.2 Market Scenario - United States
15.2.1 Glycogen Storage Disease - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Glycogen Storage Disease - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.2.3 Glycogen Storage Disease - Access and Reimbursement Overview
15.3 Market Scenario - Germany
15.3.1 Glycogen Storage Disease - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Glycogen Storage Disease - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Glycogen Storage Disease - Access and Reimbursement Overview
15.4 Market Scenario - France
15.4.1 Glycogen Storage Disease - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Glycogen Storage Disease - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapie (2024-2034)
15.4.3 Glycogen Storage Disease - Access and Reimbursement Overview
15.5 Market Scenario - United Kingdom
15.5.1 Glycogen Storage Disease - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Glycogen Storage Disease - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Glycogen Storage Disease - Access and Reimbursement Overview
15.6 Market Scenario - Italy
15.6.1 Glycogen Storage Disease - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Glycogen Storage Disease - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies(2024-2034)
15.6.3 Glycogen Storage Disease - Access and Reimbursement Overview
15.7 Market Scenario - Spain
15.7.1 Glycogen Storage Disease - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Glycogen Storage Disease - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Glycogen Storage Disease - Access and Reimbursement Overview
15.8 Market Scenario - Japan
15.8.1 Glycogen Storage Disease - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast(2024-2034)
15.8.2 Glycogen Storage Disease - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Glycogen Storage Disease - Access and Reimbursement Overview
16 Glycogen Storage Disease - Recent Events and Inputs From Key Opinion Leaders
17 Glycogen Storage Disease Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 Glycogen Storage Disease Market - Strategic Recommendations19 Appendix

Methodology

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