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Sandhoff Disease Market Report and Forecast 2024-2032
Market Report (7 days) I 2024-02-09 I 180 Pages I EMR Inc.
Sandhoff Disease Market Report and Forecast 2024-2032
Sandhoff Disease Market Outlook
The Sandhoff disease market size was valued at USD 10.18 billion in 2023, driven growing interest in gene therapy as a potential curative approach across the major markets. The market size is anticipated to grow at a CAGR of 5.87% during the forecast period of 2024-2032 and attain a value of USD 17 billion by 2032.
Sandhoff Disease: Introduction
Sandhoff disease is a rare, inherited genetic disorder characterized by the harmful accumulation of certain fats (lipids) in the brain and other organs. It's caused by mutations in the HEXB gene, leading to a deficiency of the enzymes beta-hexosaminidase A and B. This deficiency results in progressive damage to the central nervous system. Symptoms appearing in infancy include motor skill deterioration, muscle weakness, and developmental delays. Sandhoff disease is severe, typically leading to early childhood mortality.
Key Trends in the Sandhoff Disease Market
Ongoing research efforts are centered on developing enzyme replacement therapies to compensate for the deficient enzymes in affected individuals, aiming to slow disease progression.
There's a growing interest in gene therapy as a potential curative approach, with research directed at correcting the underlying genetic mutations causing the enzyme deficiencies.
As a definitive cure is still under research, the market sees a continuous demand for supportive care products and services aimed at managing symptoms and improving the quality of life for patients. This includes mobility aids, respiratory support, and nutritional products.
Patient advocacy groups are increasingly active in raising awareness about Sandhoff disease, supporting affected families, and promoting research funding.
Recognizing the need for treatments for rare diseases like Sandhoff disease, regulatory agencies offer incentives such as longer periods of exclusivity, tax credits, and assistance with clinical trial design to encourage the development of orphan drugs.
Collaboration between biotech firms, academic institutions, and healthcare organizations is key in driving advancements in the understanding and treatment of Sandhoff disease, often supported by government grants and private investments.
Sandhoff Disease Market Segmentation
Market Breakup by Type
- Infantile
- Juvenile
- Late Onset
Market Breakup by Therapy
- Gene Therapy
- Enzyme Replacement Therapy
- Stem Cell Therapy
- Others
Market Breakup by Treatment
- Medication
- Surgery
Market Breakup by Drugs
- Market Overview
- Anticonvulsants
- Miglustat
- Others
Market Breakup by Route of Administration
- Oral
- Inhalation
- Parenteral
- Others
Market Breakup by Distribution Channel
- Hospital Pharmacy
- Retail Pharmacy
Market Breakup by Region
- United States
- EU-4 and the United Kingdom
o Germany
o France
o Italy
o Spain
o United Kingdom
- Japan
Sandhoff Disease Market Overview
In North America, the Sandhoff disease market is focused on research and the development of treatment options due to the rarity and severity of the condition. Efforts are directed towards gene therapy, enzyme replacement therapies, and other innovative approaches to manage and treat the disease. The market is also influenced by strong patient advocacy groups and support networks that drive awareness, fund research, and provide resources for affected families.
Europe's market is characterized by comprehensive healthcare systems and strong regulatory support for rare disease research and treatment. The European Union has initiatives like the Orphan Drug Regulation, which encourages the development of treatments for rare diseases, including Sandhoff disease. Collaborative research across EU countries is a key factor, with a focus on developing gene therapies and understanding the disease's pathophysiology to improve patient outcomes.
Japan's market for Sandhoff disease is influenced by advanced healthcare research and a commitment to innovative treatment solutions. The country has specific programs and policies to address rare diseases, providing support for research and development of new treatments. Efforts in Japan are geared towards early diagnosis and the exploration of novel therapies, including stem cell research and enzyme replacement therapies, to provide care and potentially extend the life expectancy of those affected by the disease.
Sandhoff Disease Market: Competitor Landscape
The key features of the market report include patent analysis, grants analysis, clinical trials analysis, funding and investment analysis, partnerships, and collaborations analysis by the leading key players.
- NeoImmuneTech
- Neurimmune
- GlaxoSmithKline Plc
- Sanofi SA
- Gilead Sciences
- Allergen Plc
- Novatris AG
- Abbvie Inc.
- Bristol-Myers Squibb Company
- Akero Therapeutics, Inc.
- Bristol-Myers Squibb Company
- Alexion Pharmaceuticals, Inc
- Grifols
- Intellia Therapeutics
- Ionis Pharmaceuticals, Inc.
We at Expert Market Research always strive to provide you with the latest information. The numbers in the article are only indicative and may be different from the actual report.
1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage - Key Segmentation and Scope
1.4 Research Methodology
2 Executive Summary
3 Sandhoff Disease Overview
3.1 Guidelines and Stages
3.2 Pathophysiology
3.3 Screening and Diagnosis
3.4 Treatment Pathway
4 Patient Profile
4.1 Patient Profile Overview
4.2 Patient Psychology and Emotional Impact Factors
4.3 Risk Assessment and Treatment Success Rate
5 Sandhoff Disease Epidemiology Analysis - 7MM
5.1 7MM Epidemiology Scenario Overview (2017-2032)
5.2 United States Sandhoff Disease Epidemiology Forecast (2017-2032)
5.3 EU-4 and United Kingdom Sandhoff Disease Epidemiology Forecast (2017-2032)
5.3.1 Germany Sandhoff Disease Epidemiology Forecast (2017-2032)
5.3.2 France Sandhoff Disease Epidemiology Forecast (2017-2032)
5.3.3 Italy Sandhoff Disease Epidemiology Forecast (2017-2032)
5.3.4 Spain Sandhoff Disease Epidemiology Forecast (2017-2032)
5.3.5 United Kingdom Sandhoff Disease Epidemiology Forecast (2017-2032)
5.4 Japan Sandhoff Disease Epidemiology Forecast (2017-2032)
6 Sandhoff Disease Market Overview - 7MM
6.1 Sandhoff Disease Market Historical Value (2017-2023)
6.2 Sandhoff Disease Market Forecast Value (2024-2032)
7 Sandhoff Disease Market Landscape - 7MM
7.1 Sandhoff Disease Developers Landscape
7.1.1 Analysis by Year of Establishment
7.1.2 Analysis by Company Size
7.1.3 Analysis by Region
7.2 Sandhoff Disease Product Landscape
7.2.1 Analysis by Diseases Type
7.2.2 Analysis by Treatments
7.2.3 Analysis by Route of Administration
8 Sandhoff Disease Challenges and Unmet Needs
8.1 Treatment Pathway Challenges
8.2 Compliance and Drop-Out Analysis
8.3 Awareness and Prevention Gaps
9 Cost of Treatment
10 Sandhoff Disease Market Dynamics
10.1 Market Drivers and Constraints
10.2 SWOT Analysis
10.3 Porter's Five Forces Model
10.4 Key Demand Indicators
10.5 Key Price Indicators
10.6 Industry Events, Initiatives, and Trends
10.7 Value Chain Analysis
11 Sandhoff Disease Market Segmentation - 7MM
11.1 Sandhoff Disease Market by Type
11.1.1 Market Overview
11.1.2 Infantile
11.1.3 Juvenile
11.1.4 Late Onset
11.2 Sandhoff Disease Market by Therapy
11.2.1 Market Overview
11.2.2 Gene Therapy
11.2.3 Enzyme Replacement Therapy
11.2.4 Stem Cell Therapy
11.2.5 Others
11.3 Sandhoff Disease Market by Treatment
11.3.1 Market Overview
11.3.2 Medication
11.3.3 Surgery
11.4 Sandhoff Disease Market by Drugs
11.4.1 Market Overview
11.4.2 Anticonvulsants
11.4.3 Miglustat
11.4.4 Others
11.5 Sandhoff Disease Market by Route of Administration
11.5.1 Market Overview
11.5.2 Oral
11.5.3 Inhalation
11.5.4 Parenteral
11.5.5 Others
11.6 Sandhoff Disease Market by Distribution Channel
11.6.1 Market Overview
11.6.2 Hospital Pharmacy
11.6.3 Retail Pharmacy
11.7 Sandhoff Disease Market by Region
11.7.1 Market Overview
11.7.2 United States
11.7.3 EU-4 and the United Kingdom
11.7.3.1 Germany
11.7.3.2 France
11.7.3.3 Italy
11.7.3.4 Spain
11.7.3.5 United Kingdom
11.7.4 Japan
12 United States Sandhoff Disease Market
12.1 Sandhoff Disease Market Historical Value (2017-2023)
12.2 Sandhoff Disease Market Forecast Value (2024-2032)
12.2.1 Sandhoff Disease Market by Disease Type
12.2.2 Sandhoff Disease Market by Treatment Type
13 EU-4 and United Kingdom Sandhoff Disease Market
13.1 Sandhoff Disease Market Historical Value (2017-2023)
13.2 Sandhoff Disease Market Forecast Value (2024-2032)
13.3 Germany Sandhoff Disease Market Overview
13.3.1 Sandhoff Disease Market by Disease Type
13.3.2 Sandhoff Disease Market by Treatment Type
13.4 France Sandhoff Disease Market Overview
13.4.1 Sandhoff Disease Market by Disease Type
13.4.2 Sandhoff Disease Market by Treatment Type
13.5 Italy Sandhoff Disease Market Overview
13.5.1 Sandhoff Disease Market by Disease Type
13.5.2 Sandhoff Disease Market by Treatment Type
13.6 Spain Sandhoff Disease Market Overview
13.6.1 Sandhoff Disease Market by Disease Type
13.6.2 Sandhoff Disease Market by Treatment Type
13.7 United Kingdom Sandhoff Disease Market Overview
13.7.1 Sandhoff Disease Market by Disease Type
13.7.2 Sandhoff Disease Market by Treatment Type
14 Japan Sandhoff Disease Market
14.1 Sandhoff Disease Market Historical Value (2017-2023)
14.2 Sandhoff Disease Market Forecast Value (2024-2032)
14.3 Sandhoff Disease Market by Disease Type
15 Patent Analysis
15.1 Analysis by Type of Patent
15.2 Analysis by Publication year
15.3 Analysis by Issuing Authority
15.4 Analysis by Patent Age
15.5 Analysis by CPC Analysis
15.6 Analysis by Patent Valuation
15.7 Analysis by Key Players
16 Grants Analysis
16.1 Analysis by year
16.2 Analysis by Amount Awarded
16.3 Analysis by Issuing Authority
16.4 Analysis by Grant Application
16.5 Analysis by Funding Institute
16.6 Analysis by NIH Departments
16.7 Analysis by Recipient Organization
17 Clinical Trials Analysis
17.1 Analysis by Trial Registration Year
17.2 Analysis by Trial Status
17.3 Analysis by Trial Phase
17.4 Analysis by Therapeutic Area
17.5 Analysis by Geography
18 Funding Analysis
18.1 Analysis by Funding Instances
18.2 Analysis by Type of Funding
18.3 Analysis by Funding Amount
18.4 Analysis by Leading Players
18.5 Analysis by Leading Investors
18.6 Analysis by Geography
19 Partnership and Collaborations Analysis
19.1 Analysis by Partnership Instances
19.2 Analysis by Type of Partnership
19.3 Analysis by Leading Players
19.4 Analysis by Geography
20 Supplier Landscape
20.1 NeoImmuneTech
20.1.1 Financial Analysis
20.1.2 Product Portfolio
20.1.3 Demographic Reach and Achievements
20.1.4 Mergers and Acquisition
20.1.5 Certifications
20.2 Neurimmune
20.2.1 Financial Analysis
20.2.2 Product Portfolio
20.2.3 Demographic Reach and Achievements
20.2.4 Mergers and Acquisition
20.2.5 Certifications
20.3 GlaxoSmithKline Plc
20.3.1 Financial Analysis
20.3.2 Product Portfolio
20.3.3 Demographic Reach and Achievements
20.3.4 Mergers and Acquisition
20.3.5 Certifications
20.4 Sanofi SA
20.4.1 Financial Analysis
20.4.2 Product Portfolio
20.4.3 Demographic Reach and Achievements
20.4.4 Mergers and Acquisition
20.4.5 Certifications
20.5 Gilead Sciences
20.5.1 Financial Analysis
20.5.2 Product Portfolio
20.5.3 Demographic Reach and Achievements
20.5.4 Mergers and Acquisition
20.5.5 Certifications
20.6 Allergen Plc
20.6.1 Financial Analysis
20.6.2 Product Portfolio
20.6.3 Demographic Reach and Achievements
20.6.4 Mergers and Acquisition
20.6.5 Certifications
20.7 Novatris AG
20.7.1 Financial Analysis
20.7.2 Product Portfolio
20.7.3 Demographic Reach and Achievements
20.7.4 Mergers and Acquisition
20.7.5 Certifications
20.8 Abbvie Inc.
20.8.1 Financial Analysis
20.8.2 Product Portfolio
20.8.3 Demographic Reach and Achievements
20.8.4 Mergers and Acquisition
20.8.5 Certifications
20.9 Bristol-Myers Squibb Company
20.9.1 Financial Analysis
20.9.2 Product Portfolio
20.9.3 Demographic Reach and Achievements
20.9.4 Mergers and Acquisition
20.9.5 Certifications
20.10 Akero Therapeutics, Inc.
20.10.1 Financial Analysis
20.10.2 Product Portfolio
20.10.3 Demographic Reach and Achievements
20.10.4 Mergers and Acquisition
20.10.5 Certifications
20.11 Alexion Pharmaceuticals, Inc
20.11.1 Financial Analysis
20.11.2 Product Portfolio
20.11.3 Demographic Reach and Achievements
20.11.4 Mergers and Acquisition
20.11.5 Certifications
20.12 Grifols
20.12.1 Financial Analysis
20.12.2 Product Portfolio
20.12.3 Demographic Reach and Achievements
20.12.4 Mergers and Acquisition
20.12.5 Certifications
20.13 Intellia Therapeutics
20.13.1 Financial Analysis
20.13.2 Product Portfolio
20.13.3 Demographic Reach and Achievements
20.13.4 Mergers and Acquisition
20.13.5 Certifications
20.14 Ionis Pharmaceuticals, Inc.
20.14.1 Financial Analysis
20.14.2 Product Portfolio
20.14.3 Demographic Reach and Achievements
20.14.4 Mergers and Acquisition
20.14.5 Certifications
21 Sandhoff Disease Market - Distribution Model (Additional Insight)
21.1 Overview
21.2 Potential Distributors
21.3 Key Parameters for Distribution Partner Assessment
22 Key Opinion Leaders (KOL) Insights (Additional Insight)
23 Company Competitiveness Analysis (Additional Insight)
23.1 Very Small Companies
23.2 Small Companies
23.3 Mid-Sized Companies
23.4 Large Companies
23.5 Very Large Companies
24 Payment Methods (Additional Insight)
24.1 Government Funded
24.2 Private Insurance
24.3 Out-of-Pocket
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