GM1 Gangliosidosis Market: Epidemiology, Therapies, Companies - DelveInsight | Featuring Passage Bio Inc., Gain Therapeutics, Sio Gene Therapies

GM1 Gangliosidosis is a rare lysosomal storage disorder with an incidence of 1 in 100,000-200,000 live births, classified into infantile (0-6 months), late infantile (7 months-5 years), and adult-onset (6+ years) forms based on age at onset.

Therapies for GM1 Gangliosidosis, including PBGM01 and others, are expected to drive growth in the GM1 Gangliosidosis market over the coming years.

DelveInsight has released a new report titled "GM1 Gangliosidosis - Market Insights, Epidemiology, and Market Forecast-2034," offering a comprehensive analysis of GM1 Gangliosidosis. The report covers historical and projected epidemiology, as well as market trends in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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Some of the key facts of the GM1 Gangliosidosis Market Report:

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The GM1 Gangliosidosis market is anticipated to grow steadily, driven by ongoing research, increasing awareness that boosts diagnosis rates, and the development of potential disease-modifying therapies.

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Among the GM1 Gangliosidosis subtypes, Type 1 has the highest incidence, primarily due to its severe clinical presentation, early symptom onset-usually around six months of age-and significant impact, which leads to more frequent detection and diagnosis.

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Currently, no approved therapies exist for GM1 Gangliosidosis, and the pipeline of new treatments remains limited, posing a major challenge for patients. A promising avenue of research is gene therapy, which targets the underlying genetic cause of GM1 Gangliosidosis and holds potential as a transformative approach for managing this rare disorder.

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GM1 Gangliosidosis is a rare inherited lysosomal storage disorder passed down in an autosomal recessive manner, affecting approximately 1 in 100,000 to 200,000 live births globally. In France, the estimated incidence is around 1 in 210,000 live births.

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Leading companies, including Passage Bio Inc., Gain Therapeutics, Sio Gene Therapies, and others, are actively developing new therapies to improve the GM1 Gangliosidosis treatment landscape. Promising candidates under investigation include PBGM01 and several others.

GM1 Gangliosidosis Overview

The diagnosis of GM1 Gangliosidosis generally involves a combination of clinical evaluation, genetic testing, and biochemical analysis. Key signs include developmental delays, seizures, and other neurological symptoms. Genetic testing identifies mutations in the GLB1 gene, while biochemical assays confirm the diagnosis by detecting beta-galactosidase deficiency and elevated GM1 ganglioside levels in tissues or body fluids.

Treatment for GM1 Gangliosidosis primarily focuses on symptom management and supportive care. Interventions such as physical, occupational, and speech therapy can help address developmental challenges and improve quality of life. Additional support may include anti-seizure medications, respiratory support, and nutritional management to address specific symptoms and complications.

Currently, there is no cure for GM1 Gangliosidosis. However, several experimental therapies-including gene therapy and enzyme replacement therapy-are being investigated in preclinical and clinical studies. These approaches aim to correct the underlying genetic defect and potentially slow disease progression. While promising, these therapies are still in early development, and further research is required to assess their safety and efficacy in patients.

GM1 Gangliosidosis Market Outlook

Current management of GM1 gangliosidosis primarily revolves around symptom relief and supportive care, including physical therapy, anti-seizure medications, nutritional support via gastrostomy, and airway management. While these interventions help improve quality of life, they do not target the underlying genetic defect or significantly alter disease progression. At present, there are no FDA-approved therapies that directly treat GM1 gangliosidosis.

Encouraging developments are emerging, however. Gene therapy is being investigated as a potential treatment by delivering a functional copy of the defective gene to address the root cause of the disorder. Similarly, enzyme replacement therapy (ERT), which provides the missing beta-galactosidase enzyme, is under study to reduce toxic ganglioside accumulation and slow neurodegeneration.

These investigational, disease-modifying therapies hold promise for improving long-term outcomes and could represent a transformative shift in GM1 gangliosidosis treatment. As research advances, the GM1 gangliosidosis market in the seven major markets (7MM) is expected to undergo significant changes between 2020 and 2034.

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GM1 Gangliosidosis Emerging Drugs

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PBGM01: Passage Bio, Inc.

Scope of the GM1 Gangliosidosis Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key GM1 Gangliosidosis Companies: Passage Bio Inc., Gain Therapeutics, Sio Gene Therapies, and others

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Key GM1 Gangliosidosis Therapies: PBGM01, and others

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GM1 Gangliosidosis Therapeutic Assessment: GM1 Gangliosidosis current marketed and GM1 Gangliosidosis emerging therapies

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GM1 Gangliosidosis Market Dynamics: GM1 Gangliosidosis market drivers and GM1 Gangliosidosis market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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GM1 Gangliosidosis Unmet Needs, KOL's views, Analyst's views, GM1 Gangliosidosis Market Access and Reimbursement

To know what's more in our GM1 Gangliosidosis report, visit https://www.delveinsight.com/report-store/gm1-gangliosidosis-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

Key benefits of the GM1 Gangliosidosis Market Report:

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GM1 Gangliosidosis market report covers a descriptive overview and comprehensive insight of the GM1 Gangliosidosis Epidemiology and GM1 Gangliosidosis market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).

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The GM1 Gangliosidosis market report provides insights into the current and emerging therapies.

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The GM1 Gangliosidosis market report provides a global historical and forecasted market covering drug outreach in 7MM.

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The GM1 Gangliosidosis market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the GM1 Gangliosidosis market.

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Table of Contents

1. Report Introduction

2. Executive Summary

3. SWOT analysis

4. GM1 Gangliosidosis Patient Share (%) Overview at a Glance

5. GM1 Gangliosidosis Market Overview at a Glance

6. GM1 Gangliosidosis Disease Background and Overview

7. GM1 Gangliosidosis Epidemiology and Patient Population

8. Country-Specific Patient Population of GM1 Gangliosidosis

9. GM1 Gangliosidosis Current Treatment and Medical Practices

10. Unmet Needs

11. GM1 Gangliosidosis Emerging Therapies

12. GM1 Gangliosidosis Market Outlook

13. Country-Wise GM1 Gangliosidosis Market Analysis (2020-2034)

14. Market Access and Reimbursement of Therapies

15. Market drivers

16. Market barriers

17. Appendix

18. GM1 Gangliosidosis Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

Click here to read more about GM1 Gangliosidosis Market Outlook 2034 [https://www.delveinsight.com/report-store/gm1-gangliosidosis-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Related Reports:

GM1 Gangliosidosis Pipeline Insights, DelveInsight

"GM1 Gangliosidosis Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the GM1 Gangliosidosis market. A detailed picture of the GM1 Gangliosidosis pipeline landscape is provided, which includes the disease overview and GM1 Gangliosidosis treatment guidelines.

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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