Glycogen Storage Disease Type III Market: By Disease Type (glycogen storage disease type III Type A, glycogen storage disease type III Type B, Others), By Treatment (Dietary Management, Supportive Care, Enzyme Replacement Therapy, Gene Therapy), By End-user (Hospitals, Specialty Clinics, Research Institutes), and Geography

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Glycogen storage disease type III Market size was valued at USD XX million in 2023 and is expected to grow at an XX% CAGR from 2024-2030. Glycogen Storage Disease Type III, also known as Cori disease or Forbes disease, is a rare inherited metabolic disorder characterized by the body's inability to properly break down glycogen. This condition results from mutations in the AGL gene, which provides instructions for producing the glycogen debranching enzyme. Recent statistics indicate that glycogen storage disease type III affects approximately 1 in 100,000 individuals worldwide, with higher prevalence in certain populations such as North African Jews, where incidence rates can reach 1 in 5,400. The market has seen a surge in research activities, with the number of clinical trials for glycogen storage disease type III treatments increasing in past five years. Key drivers of the glycogen storage disease type III market include rising diagnosis rates, improved genetic testing capabilities, and growing patient advocacy efforts. The development of enzyme replacement therapies and gene therapies presents significant opportunities for market growth. However, the market faces challenges such as the high cost of treatment and limited awareness among healthcare professionals in some regions.

Current trends in the glycogen storage disease type III market include a focus on personalized medicine approaches, increased collaboration between pharmaceutical companies and research institutions, and the exploration of combination therapies. The market has also witnessed a growing emphasis on improving quality of life for patients through better management strategies and supportive care. In recent years, there has been a notable increase in funding for rare disease research, with glycogen storage disease type III benefiting from initiatives such as the NIH's Rare Diseases Clinical Research Network. This has led to a 25% increase in publications related to glycogen storage disease type III over the past three years, indicating growing scientific interest and potential for new therapeutic developments. The COVID-19 pandemic has had a mixed impact on the glycogen storage disease type III market. While it initially caused disruptions in clinical trials and patient care, it has also accelerated the adoption of telemedicine and remote monitoring technologies, potentially improving access to specialized care for glycogen storage disease type III patients in remote areas. 

Glycogen storage disease type III Market Recent Market Developments:
  • In March 2023, Ultragenyx Pharmaceutical Inc. announced positive Phase 2 results for UX053, an mRNA-based enzyme replacement therapy for glycogen storage disease type III.
  • In November 2023, Spark Therapeutics (Roche) initiated a Phase 1/2 clinical trial for SPK-3006, a gene therapy candidate for glycogen storage disease type III.

Glycogen Storage Disease Type III Market Summary

Study Period

2024-2030

Base Year

2023

CAGR

X%

Largest Market

North America

Fastest Growing Market

Asia Pacific

Glycogen Storage Disease Type III Market Dynamics

The Glycogen Storage Disease Type III market is propelled by dynamics such as increasing awareness and diagnosis rates, and advancements in research and development. Growing awareness, driven by patient advocacy groups like the Association for Glycogen Storage Disease, has led to a 40% increase in member engagement over five years. Improved genetic testing, particularly next-generation sequencing, has boosted diagnosis accuracy to over 95% for AGL gene mutations. Simultaneously, R&D advancements have significantly impacted the glycogen storage disease type III market. Clinical trials for glycogen storage disease type III treatments have increased by 30% in the past three years, as evidenced by clinicaltrials.gov registrations. Pharmaceutical companies are investing in novel approaches such as enzyme replacement and gene therapies, with promising preclinical results for mRNA-based treatments. These dynamics are creating a robust pipeline of potential therapies, addressing the unmet medical needs of patients, and driving glycogen storage disease type III market growth despite the condition's rarity.

Glycogen Storage Disease Type III Market Segmentation

By Disease type
  • glycogen storage disease type III Type A
  • glycogen storage disease type III Type B
  • Others
By Treatment
  • Dietary Management
  • Supportive Care
  • Enzyme Replacement Therapy
  • Gene Therapy
By End User
  • Hospitals
  • Specialty Clinics
  • Research Institutes
Geography
  • North America
  • Europe
  • The Asia Pacific
  • Latin America
  • MEA

Frequently Asked Questions

Pfizer Inc., Novo Nordisk A/S, Amicus Therapeutics, Inc., and Regeneron Pharmaceuticals, Inc. are some of the major players operating in the glycogen storage disease type III market.

Asia Pacific is estimated to grow at the highest CAGR over the forecast period.

North America accounts for the largest market share and is expected to be the primary market driver in the glycogen storage disease type III market.

Key challenges include the rarity of the disease, limited understanding of its long-term progression, and difficulties in conducting large-scale clinical trials due to the small patient population.

Key Features of the Report

  • The glycogen storage disease type III market report provides granular level information about the market size, regional market share, historic market (2019-2023), and forecast (2024-2030).
  • The report covers in-detail insights about the competitor’s overview, company share analysis, key market developments, and their key strategies.
  • The report outlines drivers, restraints, unmet needs, and trends that are currently affecting the market.
  • The report tracks recent innovations, key developments, and start-up details that are actively working in the market.
  • The report provides a plethora of information about market entry strategies, regulatory framework, and reimbursement scenarios.
1.Executive Summary
2.Global Glycogen Storage Disease Type III Market Introduction 
2.1.Global Glycogen Storage Disease Type III Market  - Taxonomy
2.2.Global Glycogen Storage Disease Type III Market  - Definitions
2.2.1.Disease type
2.2.2.Treatment
2.2.3.End User
2.2.4.Region
3.Global Glycogen Storage Disease Type III Market Dynamics
3.1. Drivers
3.2. Restraints
3.3. Opportunities/Unmet Needs of the Market
3.4. Trends
3.5. Product Landscape
3.6. New Product Launches
3.7. Impact of COVID 19 on Market
4.Global Glycogen Storage Disease Type III Market Analysis, 2019 - 2023 and Forecast 2024 - 2030
4.1.  Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
4.2.  Year-Over-Year (Y-o-Y) Growth Analysis (%) 
4.3.  Market Opportunity Analysis 
5.Global Glycogen Storage Disease Type III Market  By Disease type, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
5.1. glycogen storage disease type III Type A
5.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.1.3. Market Opportunity Analysis 
5.2. glycogen storage disease type III Type B
5.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.2.3. Market Opportunity Analysis 
5.3. Others
5.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.3.3. Market Opportunity Analysis 
6.Global Glycogen Storage Disease Type III Market  By Treatment, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
6.1. Dietary Management
6.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.1.3. Market Opportunity Analysis 
6.2. Supportive Care
6.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.2.3. Market Opportunity Analysis 
6.3. Enzyme Replacement Therapy
6.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.3.3. Market Opportunity Analysis 
6.4. Gene Therapy
6.4.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.4.3. Market Opportunity Analysis 
7.Global Glycogen Storage Disease Type III Market  By End User, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
7.1. Hospitals
7.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.1.3. Market Opportunity Analysis 
7.2. Specialty Clinics
7.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.2.3. Market Opportunity Analysis 
7.3. Research Institutes
7.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.3.3. Market Opportunity Analysis 
8.Global Glycogen Storage Disease Type III Market  By Region, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
8.1. North America
8.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.1.3. Market Opportunity Analysis 
8.2. Europe
8.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.2.3. Market Opportunity Analysis 
8.3. Asia Pacific (APAC)
8.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.3.3. Market Opportunity Analysis 
8.4. Middle East and Africa (MEA)
8.4.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.4.3. Market Opportunity Analysis 
8.5. Latin America
8.5.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.5.3. Market Opportunity Analysis 
9.North America Glycogen Storage Disease Type III Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
9.1. Disease type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.1.1.glycogen storage disease type III Type A
9.1.2.glycogen storage disease type III Type B
9.1.3.Others
9.2.  Treatment Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.2.1.Dietary Management
9.2.2.Supportive Care
9.2.3.Enzyme Replacement Therapy
9.2.4.Gene Therapy
9.3.  End User Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.3.1.Hospitals
9.3.2.Specialty Clinics
9.3.3.Research Institutes
9.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.4.1.United States of America (USA)
9.4.2.Canada
10.Europe Glycogen Storage Disease Type III Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
10.1. Disease type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.1.1.glycogen storage disease type III Type A
10.1.2.glycogen storage disease type III Type B
10.1.3.Others
10.2.  Treatment Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.2.1.Dietary Management
10.2.2.Supportive Care
10.2.3.Enzyme Replacement Therapy
10.2.4.Gene Therapy
10.3.  End User Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.3.1.Hospitals
10.3.2.Specialty Clinics
10.3.3.Research Institutes
10.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.4.1.Germany
10.4.2.France
10.4.3.Italy
10.4.4.United Kingdom (UK)
10.4.5.Spain
11.Asia Pacific (APAC) Glycogen Storage Disease Type III Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
11.1. Disease type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.1.1.glycogen storage disease type III Type A
11.1.2.glycogen storage disease type III Type B
11.1.3.Others
11.2.  Treatment Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.2.1.Dietary Management
11.2.2.Supportive Care
11.2.3.Enzyme Replacement Therapy
11.2.4.Gene Therapy
11.3.  End User Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.3.1.Hospitals
11.3.2.Specialty Clinics
11.3.3.Research Institutes
11.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.4.1.China
11.4.2.India
11.4.3.Australia and New Zealand (ANZ)
11.4.4.Japan
11.4.5.Rest of APAC
12.Middle East and Africa (MEA) Glycogen Storage Disease Type III Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
12.1. Disease type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.1.1.glycogen storage disease type III Type A
12.1.2.glycogen storage disease type III Type B
12.1.3.Others
12.2.  Treatment Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.2.1.Dietary Management
12.2.2.Supportive Care
12.2.3.Enzyme Replacement Therapy
12.2.4.Gene Therapy
12.3.  End User Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.3.1.Hospitals
12.3.2.Specialty Clinics
12.3.3.Research Institutes
12.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.4.1.GCC Countries
12.4.2.South Africa
12.4.3.Rest of MEA
13.Latin America Glycogen Storage Disease Type III Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
13.1. Disease type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
13.1.1.glycogen storage disease type III Type A
13.1.2.glycogen storage disease type III Type B
13.1.3.Others
13.2.  Treatment Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
13.2.1.Dietary Management
13.2.2.Supportive Care
13.2.3.Enzyme Replacement Therapy
13.2.4.Gene Therapy
13.3.  End User Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
13.3.1.Hospitals
13.3.2.Specialty Clinics
13.3.3.Research Institutes
13.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
13.4.1.Brazil
13.4.2.Mexico
13.4.3.Rest of LA
14. Competition Landscape
14.1.  Market Player Profiles (Introduction, Brand/Product Sales, Financial Analysis, Product Offerings, Key Developments, Collaborations, M & A, Strategies, and SWOT Analysis) 
14.2.1.Ultragenyx Pharmaceutical Inc.
14.2.2.Alexion Pharmaceuticals Inc.
14.2.3.AstraZeneca
14.2.4.Sanofi S.A.
14.2.5.Takeda Pharmaceutical Company Limited
14.2.6.BioMarin Pharmaceutical Inc.
14.2.7.Spark Therapeutics
14.2.8.Roche
14.2.9.Pfizer Inc.
14.2.10.Novo Nordisk A/S
14.2.11.Amicus Therapeutics, Inc.
14.2.12.Regeneron Pharmaceuticals, Inc.
14.2.13.Vertex Pharmaceuticals Incorporated
14.2.14.Genzyme Corporation
14.2.15.Sanofi
14.2.16.Shire Pharmaceuticals
14.2.17.Takeda
14.2.18.Abeona Therapeutics Inc.
15. Research Methodology 
16. Appendix and Abbreviations 
  • Ultragenyx Pharmaceutical Inc.
  • Alexion Pharmaceuticals Inc.
  • AstraZeneca
  • Sanofi S.A.
  • Takeda Pharmaceutical Company Limited
  • BioMarin Pharmaceutical Inc.
  • Spark Therapeutics
  • Roche
  • Pfizer Inc.
  • Novo Nordisk A/S
  • Amicus Therapeutics, Inc.
  • Regeneron Pharmaceuticals, Inc.
  • Vertex Pharmaceuticals Incorporated
  • Genzyme Corporation
  • Sanofi
  • Shire Pharmaceuticals
  • Takeda
  • Abeona Therapeutics Inc.

Adjacent Markets