- Dietary Management
- Medication
- Gene Therapy
- Liver Transplantation
Glycogen Storage Disease Type Ia Treatment Market size was valued at USD 223.8 million in 2023 and is poised to grow at a CAGR of 5.6% from 2024 to 2030. Glycogen storage disease type Ia, also known as von Gierke disease, is a rare genetic disorder affecting glucose metabolism. The market's growth is primarily driven by increasing awareness, advancements in diagnostic techniques, and the development of novel treatment approaches. In 2022, a breakthrough in gene therapy for glycogen storage disease type Ia showed promising results in preclinical studies, potentially offering a long-term solution for patients. This development has spurred increased investment in genetic approaches to glycogen storage disease type Ia treatment. Additionally, improved dietary management strategies and the introduction of modified cornstarch products have enhanced day-to-day disease control for many patients. Government initiatives supporting rare disease research have played a crucial role in market growth.
For instance, the FDA's Orphan Drug Designation program has incentivized pharmaceutical companies to invest in glycogen storage disease type Ia treatment development. Patient advocacy groups, such as the Association for Glycogen Storage Disease, have been instrumental in raising awareness and supporting research efforts. However, the market faces challenges such as the high cost of treatment development for a small patient population and difficulties in conducting large-scale clinical trials. The complex nature of glycogen storage disease type Ia management, requiring lifelong dietary control and medication, also presents hurdles in treatment adherence and quality of life improvements. Ongoing research into novel therapeutic approaches, including enzyme replacement therapy and small molecule drugs targeting specific metabolic pathways, offers promising opportunities for market players. The increasing focus on addressing long-term complications of glycogen storage disease type Ia, such as hepatic adenomas and renal disease, is expected to drive innovation in comprehensive care strategies. Recent years have seen a significant focus on improving the quality of life for glycogen storage disease type Ia patients. The market has witnessed a trend towards personalized medicine, with researchers exploring targeted therapies based on individual patient genetics and metabolic profiles.
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The glycogen storage disease type Ia treatment market is primarily driven by the increasing diagnosis rates due to improved genetic testing capabilities and newborn screening programs. The development of novel therapies, particularly in gene therapy and metabolic modulation, has attracted significant investment in the field. Moreover, the rising demand for comprehensive care approaches, combining dietary management with emerging medical interventions, has expanded the market landscape. However, challenges persist in terms of treatment accessibility, especially in regions with limited specialized care centers. The high cost of potentially curative therapies and the need for lifelong management present economic hurdles for patients and healthcare systems. Despite these challenges, ongoing research into novel therapeutic approaches and potential curative treatments continue to drive market growth and offer hope for improved outcomes in glycogen storage disease type Ia patients.
The Glycogen Storage Disease Type Ia (GSDIa) Treatment Market was valued at USD 223.8 million in 2023 and is projected to grow at a CAGR of 5.6% from 2024 to 2030..
Ultragenyx Pharmaceutical Inc., Vitaflo International Ltd., Nestle Health Science, and Acer Therapeutics Inc. are some of the key players in the glycogen storage disease type Ia treatment market.
Increasing diagnosis rates through improved genetic testing, development of novel therapies including gene therapy, and growing awareness of rare metabolic disorders are some of the factors predicted to propel the growth of the glycogen storage disease type Ia treatment market.
North America is expected to dominate the glycogen storage disease type Ia treatment market due to its advanced healthcare infrastructure, high healthcare spending, and strong focus on rare disease research.
Limited patient pool, high treatment costs, challenges in developing treatments for a complex metabolic disorder, and limited accessibility in developing regions are some factors expected to limit the growth of the glycogen storage disease type Ia treatment market.
1.Executive Summary |
2.Global Glycogen Storage Disease Type Ia Treatment Market Introduction |
2.1.Global Glycogen Storage Disease Type Ia Treatment Market - Taxonomy |
2.2.Global Glycogen Storage Disease Type Ia Treatment Market - Definitions |
2.2.1.Treatment Type |
2.2.2.Route Administration |
2.2.3.End User |
2.2.4.Region |
3.Global Glycogen Storage Disease Type Ia Treatment 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 Ia Treatment 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 Ia Treatment Market By Treatment Type, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
5.1. Dietary Management |
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. Medication |
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. Gene Therapy |
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 |
5.4. Liver Transplantation |
5.4.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
5.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
5.4.3. Market Opportunity Analysis |
6.Global Glycogen Storage Disease Type Ia Treatment Market By Route Administration, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
6.1. Oral |
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. Parenteral |
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 |
7.Global Glycogen Storage Disease Type Ia Treatment 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. Homecare |
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 Ia Treatment 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 Ia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
9.1. Treatment Type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
9.1.1.Dietary Management |
9.1.2.Medication |
9.1.3.Gene Therapy |
9.1.4.Liver Transplantation |
9.2. Route Administration Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
9.2.1.Oral |
9.2.2.Parenteral |
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.Homecare |
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 Ia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
10.1. Treatment Type Analysis and Forecast by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
10.1.1.Dietary Management |
10.1.2.Medication |
10.1.3.Gene Therapy |
10.1.4.Liver Transplantation |
10.2. Route Administration Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
10.2.1.Oral |
10.2.2.Parenteral |
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.Homecare |
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 Ia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
11.1. Treatment Type Analysis and Forecast by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
11.1.1.Dietary Management |
11.1.2.Medication |
11.1.3.Gene Therapy |
11.1.4.Liver Transplantation |
11.2. Route Administration Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
11.2.1.Oral |
11.2.2.Parenteral |
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.Homecare |
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 Ia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
12.1. Treatment Type Analysis and Forecast by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
12.1.1.Dietary Management |
12.1.2.Medication |
12.1.3.Gene Therapy |
12.1.4.Liver Transplantation |
12.2. Route Administration Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
12.2.1.Oral |
12.2.2.Parenteral |
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.Homecare |
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 Ia Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
13.1. Treatment Type Analysis and Forecast by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
13.1.1.Dietary Management |
13.1.2.Medication |
13.1.3.Gene Therapy |
13.1.4.Liver Transplantation |
13.2. Route Administration Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
13.2.1.Oral |
13.2.2.Parenteral |
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.Homecare |
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.Vitaflo International Ltd. |
14.2.3.Nestle Health Science |
14.2.4.Acer Therapeutics Inc. |
14.2.5.Recordati Rare Diseases |
14.2.6.Arcturus Therapeutics Holdings Inc. |
14.2.7.Genexine, Inc. |
14.2.8.Alexion Pharmaceuticals, Inc. (now part of AstraZeneca) |
14.2.9.Nutricia North America |
14.2.10.Vitaflo USA LLC |
15. Research Methodology |
16. Appendix and Abbreviations |