Sandhoff Disease Market

Market Overview:

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?