Summary

The 7 major Sandhoff disease markets are expected to exhibit a CAGR of 3.07% during 2024-2034.

The Sandhoff disease market has been comprehensively analyzed in IMARC's new report titled "Sandhoff Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Sandhoff disease is a rare, inherited metabolic disorder that mainly affects the central nervous system. It is caused by the absence or insufficient activity of an enzyme called hexosaminidase A, which leads to the accumulation of certain fats (glycolipids) in the brain and nervous system. It usually occurs in infancy, but it can appear in children and adults too. The symptoms of this condition include abnormal bone development, awkward movements, cherry-red spots on the eyeballs, an enlarged head (macrocephaly) or enlarged liver and spleen, exaggerated reactions to loud noises, frequent respiratory infections, delayed development of motor skills, weak muscles, etc. In severe cases, infants suffering from Sandhoff disease may also experience hearing loss, paralysis, seizures, vision loss, and early death. The diagnosis of this ailment is typically made by a detailed evaluation of the patient's medical history, clinical manifestations, and physical examination. A blood workup to determine the level and activity of the hexosaminidase enzyme is also recommended. The healthcare professional may further perform genetic testing to identify gene mutations associated with the disease.

The increasing prevalence of lipid storage disorders on account of inherited deficiencies in producing functional catabolic enzymes is primarily driving the Sandhoff disease market. Apart from this, the widespread adoption of anticonvulsant drugs, including valproic acid and phenobarbital, to manage and control the symptoms of the ailment, such as seizures, is also acting as another significant growth-inducing factor. Moreover, the escalating application of substrate reduction therapy using a glucosylceramide synthase inhibitor to decrease the buildup of glycolipids in cells, which further leads to the development of the disorder, is creating a positive outlook for the market. Additionally, several key players are making extensive investments in introducing novel disease-modifying therapeutic options that slowly target the progression of the illness while maintaining adequate nutrition and hydration. This, in turn, is also propelling the market growth. Furthermore, the emerging popularity of stem cell therapy for treating the ailment, since it helps in replacing the damaged cells in the brain and nervous system to restore the function of the affected areas, is expected to drive the Sandhoff disease market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Sandhoff disease market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for Sandhoff disease and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Sandhoff disease market in any manner.

Time Period of the Study

Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034

Countries Covered

United States
Germany
France
United Kingdom
Italy
Spain
Japan

Analysis Covered Across Each Country

Historical, current, and future epidemiology scenario
Historical, current, and future performance of the Sandhoff disease market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Sandhoff disease market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Sandhoff disease marketed drugs and late-stage pipeline drugs.

In-Market Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance
Key Questions Answered in this Report:
Market Insights

How has the Sandhoff disease market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the Sandhoff disease market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the Sandhoff disease market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?

Epidemiology Insights

What is the number of prevalent cases (2018-2034) of Sandhoff disease across the seven major markets?
What is the number of prevalent cases (2018-2034) of Sandhoff disease by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Sandhoff disease by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with Sandhoff disease across the seven major markets?
What is the size of the Sandhoff disease patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of Sandhoff disease?
What will be the growth rate of patients across the seven major markets?

Sandhoff Disease: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for Sandhoff disease drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Sandhoff disease market?
What are the key regulatory events related to the Sandhoff disease market?
What is the structure of clinical trial landscape by status related to the Sandhoff disease market?
What is the structure of clinical trial landscape by phase related to the Sandhoff disease market?
What is the structure of clinical trial landscape by route of administration related to the Sandhoff disease market?

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Table of Contents

1 Preface

2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 Executive Summary

4 Sandhoff Disease - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence

5 Sandhoff Disease - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment

6 Patient Journey

7 Sandhoff Disease - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (​2018-2034​)
7.2.4 Epidemiology by Gender (​2018-2034​)
7.2.5 Diagnosed Cases (​2018-2034​)
7.2.6 Patient Pool/Treated Cases (​2018-2034​)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (​2018-2034​)
7.3.4 Epidemiology by Gender (​2018-2034​)
7.3.5 Diagnosed Cases (​2018-2034​)
7.3.6 Patient Pool/Treated Cases (​2018-2034​)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (​2018-2034​)
7.4.4 Epidemiology by Gender (​2018-2034​)
7.4.5 Diagnosed Cases (​2018-2034​)
7.4.6 Patient Pool/Treated Cases (​2018-2034​)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (​2018-2034​)
7.5.4 Epidemiology by Gender (​2018-2034​)
7.5.5 Diagnosed Cases (​2018-2034​)
7.5.6 Patient Pool/Treated Cases (​2018-2034​)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (​2018-2034​)
7.6.4 Epidemiology by Gender (​2018-2034​)
7.6.5 Diagnosed Cases (​2018-2034​)
7.6.6 Patient Pool/Treated Cases (​2018-2034​)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (​2018-2034​)
7.7.4 Epidemiology by Gender (​2018-2034​)
7.7.5 Diagnosed Cases (​2018-2034​)
7.7.6 Patient Pool/Treated Cases (​2018-2034​)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (​2018-2034​)
7.8.4 Epidemiology by Gender (​2018-2034​)
7.8.5 Diagnosed Cases (​2018-2034​)
7.8.6 Patient Pool/Treated Cases (​2018-2034​)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (​2018-2034​)
7.9.4 Epidemiology by Gender (​2018-2034​)
7.9.5 Diagnosed Cases (​2018-2034​)
7.9.6 Patient Pool/Treated Cases (​2018-2034​)

8 Sandhoff Disease - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Sandhoff Disease - Unmet Needs

10 Sandhoff Disease - Key Endpoints of Treatment

11 Sandhoff Disease - Marketed Products
11.1 List of Sandhoff Disease Marketed Drugs Across the Top 7 Markets
11.1.1 Drug Name – Company Name
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets

Kindly note that the complete list of marketed drugs has been provided in the report.

12 Sandhoff Disease - Pipeline Drugs
12.1 List of Sandhoff Disease Pipeline Drugs Across the Top 7 Markets
12.1.1 Drug Name – Company Name
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status

Kindly note that the complete list of pipeline drugs has been provided in the report.

13. Sandhoff Disease - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Sandhoff Disease – Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events

15 Sandhoff Disease - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Sandhoff Disease - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Sandhoff Disease - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Sandhoff Disease - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Sandhoff Disease - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Sandhoff Disease - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Sandhoff Disease - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Sandhoff Disease - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Sandhoff Disease - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Sandhoff Disease - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Sandhoff Disease - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Sandhoff Disease - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Sandhoff Disease - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Sandhoff Disease - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Sandhoff Disease - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Sandhoff Disease - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Sandhoff Disease - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Sandhoff Disease - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Sandhoff Disease - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Sandhoff Disease - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Sandhoff Disease - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Sandhoff Disease - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Sandhoff Disease - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Sandhoff Disease - Access and Reimbursement Overview

16 Sandhoff Disease - Recent Events and Inputs From Key Opinion Leaders

17 Sandhoff Disease Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Sandhoff Disease Market – Strategic Recommendations

19 Appendix