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Sandhoff Disease Market Size, Share, Trends, Growth, Report: By Type: Infantile, Juvenile, Late Onset; By Therapy: Gene Therapy, Enzyme Replacement Therapy, Stem Cell Therapy, Others; By Treatment: Medication, Surgery; By Drugs; By Route of Administration; By Distribution Channel; Regional Analysis; Supplier Landscape; 2024-2032
Sandhoff Disease Market Outlook
The Sandhoff disease market size was valued at USD 10.18 billion in 2023, driven by the increased competition among pharmaceutical companies across the 7 major markets. The market size is anticipated to grow at a CAGR of 5.87% during the forecast period of 2024-2032 to achieve a value of USD 17 billion by 2032.
Sandhoff Disease: Introduction
Sandhoff disease, a rare inherited disorder, gradually devastates nerve cells within the central nervous system, encompassing the brain and spinal cord. It is categorized into three primary types, distinguished by the onset of signs and symptoms: infantile, juvenile, and adult variants. Among these, the infantile manifestation stands as the most prevalent and severe, manifesting during infancy itself. Typically, infants affected by this form of the disease seem healthy until around 3 to 6 months of age, after which a noticeable decline in their developmental progress occurs, accompanied by weakening of movement-related muscles.
Sandhoff Disease Market Analysis
The market growth is driven by ongoing research and development activities that are focused on discovering the best treatment approach for this life-threatening rare genetic disease. The increasing awareness about Sandhoff disease is a major factor that individuals are now asking for effective treatment, propelling the market demand. Since the market does not have a full-proof effective treatment available, the market growth may be restrained in the forecast period.
Furthermore, the dedicated actions by the doctors and researchers are exhibiting a rigid determination toward finding a cure for this condition. Sandhoff disease is a rare disorder that slowly destroys the nerve cells in the nervous system resulting in patient losing their abilities to sit, see, and sense collectively. Children suffering from this disease often die at an age not more than three years.
Such crucial yet heartbreaking facts are prominent to consider for market players and doctors to find a solution for this rare disorder which will result in increased research and development, activities, FDA approvals, and clinical trials, expected to bolster the market growth and relieve the patients suffering from the medical condition.
Sandhoff Disease Market Segmentations
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
- 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
- Germany
- France
- Italy
- Spain
- United Kingdom
- Japan
Sandhoff Disease Market Overview
The market is expected to experience significant growth driven by factors such as increasing awareness among individuals regarding genetic disorders and the increasing allocation of special drug designations by regulatory authorities is anticipated to drive market growth. Moreover, intensifying competition among pharmaceutical companies is acting as a catalyst in boosting market growth. Nonetheless, obstacles such as limited accessibility to essential services in remote regions and insufficient training and knowledge among healthcare professionals are potentially expected to restrain growth. The increasing number of investments from the biotechnology and pharmaceutical industries into research and development initiatives are expected to bolster the Sandhoff disease market growth in the forecast period.
The United States is currently leading the market and is expected to continue dominating the market in the forecast period. The regional market growth can be attributed to advanced treatment availability and the presence of a robustly developed healthcare infrastructure. Other regions are also likely to witness significant growth in the coming years owing to its steady advancement in healthcare infrastructure and escalating governmental investments in research and development.
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. The major companies in the market are as follows:
- 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.
Please note that this only represents a partial list of companies, and the complete list has been provided in the report.
Key Highlights of the Report
| REPORT FEATURES | DETAILS |
| Base Year | 2023 |
| Historical Period | 2017-2023 |
| Forecast Period | 2024-2032 |
| Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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| Breakup by Type |
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| Breakup by Therapy |
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| Breakup by Treatment |
|
| Breakup by Drugs |
|
| Breakup by Route of Administration |
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| Breakup by Distribution Channel |
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| Breakup by Region |
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| Market Dynamics |
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| Competitive Landscape |
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| Companies Covered |
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*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR 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
*Additional insights provided are customisable as per client requirements.
Key Questions Answered in the Report
The market attained a value of about USD 10.18 billion in 2023, driven by the risen awareness.
The market is anticipated to grow at a CAGR of 5.87% during the forecast period of 2024-2032 and is likely to reach a market value of USD 17 billion by 2032.
The market growth is driven by the increased investments in the biotechnology and pharmaceutical companies, along with the increasing awareness about the condition among people.
The increased research and development activities to find a solution for this rare genetic disease is a major trend influencing the market growth.
The major regions of the market include United States, EU-4 and the United Kingdom, ad Japan. EU-4 is inclusive of Germany, France, Italy, and Spain.
Various types of diseases include infantile, juvenile, and late onset.
Types of therapies available for Sandhoff disease are gene therapy, enzyme replacement therapy, and stem cell therapy, among others.
Patients get treated with medication or surgery.
Drugs used for the treatment include anticonvulsants, and miglustat, among others.
Routes of administration include oral, inhalation, and parenteral routes.
Distribution channels are hospital pharmacies and retail pharmacies.
Key players involved in the market are 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, Ionis Pharmaceuticals, Inc., and Intellia Therapeutics.
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