Press release
GM2 Gangliosidosis Market to Witness Promising Upswing by 2034, DelveInsight Forecasts
To Know in detail about the GM2 Gangliosidosis market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; GM2 Gangliosidosis Market Forecast
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Some of the key facts of the GM2 Gangliosidosis Market Report:
• The GM2 Gangliosidosis market size is anticipated to grow with a significant CAGR during the study period (2020-2034)
• In January 2025, Azafaros B.V. announced that its lead candidate, nizubaglustat, has received orphan drug designation from regulatory authorities in both the United States and the European Union for the treatment of GM1 gangliosidosis. Additionally, the company's Clinical Trial Application (CTA) for two global Phase 3 studies assessing the drug's efficacy and safety in GM1/GM2 gangliosidoses and Niemann-Pick Type C (NPC) has been approved in multiple European countries. Azafaros plans to commence these global trials in the second quarter of 2025.
• According to the analysis, approximately seventy cases of GM2 gangliosidosis were documented in the United Kingdom, with around forty individuals diagnosed with Tay-Sachs disease, thirty with Sandhoff disease, and two with GM2 activator protein deficiency.
• According to DelveInsight's analysis, in the United States, the incidence of TSD is approximately 1 in every 320,000 live births, while the carrier frequency is around 1 in 250 individuals.
• Approximately 30 patients were identified with mutations in the HEXA gene, while around fifteen patients exhibited defects in the HEXB gene. Among Tay-Sachs patients, about twenty individuals manifested the infantile form of the disease, with the age of diagnosis spanning from 7 months to 3 years.
• Key GM2 Gangliosidosis Companies: Azafaros A.G., IntraBio, Children's National Research Institute, Sanofi, and others
• Key GM2 Gangliosidosis Therapies: AZ-3102 (nizubaglustat), IB1001, Zavesca (Miglustat), venglustat GZ402671, and others
• The GM2 Gangliosidosis market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage GM2 Gangliosidosis pipeline products will significantly revolutionize the GM2 Gangliosidosis market dynamics.
GM2 Gangliosidosis Overview
GM2 gangliosidosis is a group of rare, inherited neurodegenerative disorders caused by the accumulation of GM2 ganglioside, a type of fatty substance, in the nerve cells of the brain and spinal cord. This accumulation results from a deficiency in specific enzymes needed to break down GM2 ganglioside. GM2 gangliosidosis is divided into two main types: Tay-Sachs disease and Sandhoff disease.
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GM2 Gangliosidosis Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
GM2 Gangliosidosis Epidemiology Segmentation:
The GM2 Gangliosidosis market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
• Total Prevalence of GM2 Gangliosidosis
• Prevalent Cases of GM2 Gangliosidosis by severity
• Gender-specific Prevalence of GM2 Gangliosidosis
• Diagnosed Cases of Episodic and Chronic GM2 Gangliosidosis
Download the report to understand which factors are driving GM2 Gangliosidosis epidemiology trends @ GM2 Gangliosidosis Epidemiology Forecast
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GM2 Gangliosidosis Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the GM2 Gangliosidosis market or expected to get launched during the study period. The analysis covers GM2 Gangliosidosis market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the GM2 Gangliosidosis Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
GM2 Gangliosidosis Therapies and Key Companies
• AZ-3102 (nizubaglustat): Azafaros A.G.
• IB1001: IntraBio
• Zavesca (Miglustat): Children's National Research Institute
• venglustat GZ402671: Sanofi
Discover more about therapies set to grab major GM2 Gangliosidosis market share @ GM2 Gangliosidosis Treatment Landscape
https://www.delveinsight.com/sample-request/gm2-gangliosidosis-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
Scope of the GM2 Gangliosidosis Market Report
• Study Period: 2020-2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key GM2 Gangliosidosis Companies: Azafaros A.G., IntraBio, Children's National Research Institute, Sanofi, and others
• Key GM2 Gangliosidosis Therapies: AZ-3102 (nizubaglustat), IB1001, Zavesca (Miglustat), venglustat GZ402671, and others
• GM2 Gangliosidosis Therapeutic Assessment: GM2 Gangliosidosis current marketed and GM2 Gangliosidosis emerging therapies
• GM2 Gangliosidosis Market Dynamics: GM2 Gangliosidosis market drivers and GM2 Gangliosidosis market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• GM2 Gangliosidosis Unmet Needs, KOL's views, Analyst's views, GM2 Gangliosidosis Market Access and Reimbursement
Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Gaurav Bora
Email: info@delveinsight.com
Contact No.: +14699457679
City: 304 S. Jones Blvd #2432, Las Vegas
State: Nevada (89107)
Country: United States
Website: https://www.delveinsight.com/consulting
About DelveInsight
DelveInsight is a leading Healthcare 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.
It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.
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