GM1 Gangliosidosis Treatment Market: Advances & Emerging Therapies By 2031 | Most Leading Companies - Passage Bio, Lysogene, Sio Gene Therapies, Inc., BioStrategies LC, Sanofi, AZAFAROS, Dorphan S.A

Global GM1 Gangliosidosis Treatment Market is expected to reach at a high CAGR during the forecast period 2024-2031.

GM1 Gangliosidosis Treatment Market report, published by DataM Intelligence, provides in-depth insights and analysis on key market trends, growth opportunities, and emerging challenges. Committed to delivering actionable intelligence, DataM Intelligence empowers businesses to make informed decisions and stay ahead of the competition. Through a combination of qualitative and quantitative research methods, it offers comprehensive reports that help clients navigate complex market landscapes, drive strategic growth, and seize new opportunities in an ever-evolving global market.

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GM1 Gangliosidosis treatment focuses on managing symptoms and exploring potential curative therapies for this rare, inherited lysosomal storage disorder caused by GLB1 gene mutations. Current approaches include supportive care, enzyme replacement therapy (ERT), gene therapy, and substrate reduction therapy, aiming to slow disease progression and improve patient outcomes.

List of the Key Players in the GM1 Gangliosidosis Treatment Market:

Passage Bio, Lysogene, Sio Gene Therapies, Inc., BioStrategies LC, Sanofi, AZAFAROS, Dorphan S.A., Idorsia Pharmaceuticals Ltd, SphinCS GmbH and Viatris Inc.

Industry Development:

On August 7, 2023, Passage Bio, Inc., a clinical-stage genetic medicine company focused on developing innovative treatments for central nervous system (CNS) disorders, announced new interim safety, biomarker, and survival data from cohorts 1-4 of the Imagine-1 clinical trial. Imagine-1 is a global Phase 1/2, open-label, dose-escalation study evaluating the intra-cisternal magna (ICM) administration of AAVhu68 gene therapy PBGM01 in six cohorts of young patients with early or late infantile GM1 Gangliosidosis (GM1). GM1 is a rare and life-threatening lysosomal storage disorder caused by mutations in the GLB1 gene, resulting in severely reduced activity of the beta-galactosidase (β-Gal) enzyme.

Growth Forecast Projected:

The Global GM1 Gangliosidosis Treatment Market is anticipated to rise at a considerable rate during the forecast period, between 2024 and 2031. In 2023, the market is growing at a steady rate, and with the rising adoption of strategies by key players, the market is expected to rise over the projected horizon.

Research Process:

Both primary and secondary data sources have been used in the global GM1 Gangliosidosis Treatment Market research report. During the research process, a wide range of industry-affecting factors are examined, including governmental regulations, market conditions, competitive levels, historical data, market situation, technological advancements, upcoming developments, in related businesses, as well as market volatility, prospects, potential barriers, and challenges.

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Segment Covered in the GM1 Gangliosidosis Treatment Market:

By Type: GM1 Gangliosidosis Type 1 (Infantile), GM1 Gangliosidosis Type 2 (Juvenile), GM1 Gangliosidosis Type 3 (Adult)

By Treatment: Enzyme Replacement Therapy, Gene Therapy, Stem Cell Transplants, Substrate Reduction Therapy, Pharmacological Chaperones, Miglustat, N-Butyl-Deoxynojirimycin (NB-DNJ), Anticonvulsant Drugs

By End User: Hospitals, Ambulatory Surgical Centers, Others

Regional Analysis for GM1 Gangliosidosis Treatment Market:

The regional analysis of the GM1 Gangliosidosis Treatment Market covers key regions including North America, Europe, Asia Pacific Middle East and Africa and South America. The North America with a focus on the U.S., Canada, and Mexico; Europe, highlighting major countries like the U.K., Germany, France, and Italy, along with other nations in the region; Asia-Pacific, covering India, China, Japan, South Korea, and Australia, among others; South America, with emphasis on Colombia, Brazil, and Argentina; and the Middle East & Africa, which includes Saudi Arabia, the U.A.E., South Africa, and other countries. This comprehensive regional breakdown helps identify unique market trends and growth opportunities specific to each area.

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

Benefits of the Report:

➡ A descriptive analysis of demand-supply gap, market size estimation, SWOT analysis, PESTEL Analysis and forecast in the global market.

➡ Top-down and bottom-up approach for regional analysis

➡ Porter's five forces model gives an in-depth analysis of buyers and suppliers, threats of new entrants & substitutes and competition amongst the key market players.

➡ By understanding the value chain analysis, the stakeholders can get a clear and detailed picture of this Market

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People Also Ask:

➠ What is the global sales, production, consumption, import, and export value of the GM1 Gangliosidosis Treatment market?

➠ Who are the leading manufacturers in the global GM1 Gangliosidosis Treatment industry? What is their operational status in terms of capacity, production, sales, pricing, costs, gross margin, and revenue?

➠ What opportunities and challenges do vendors in the global GM1 Gangliosidosis Treatment industry face?

➠ Which applications, end-users, or product types are expected to see growth? What is the market share for each type and application?

➠ What are the key factors and limitations affecting the growth of the GM1 Gangliosidosis Treatment market?

➠ What are the various sales, marketing, and distribution channels in the global industry?

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