Summary

The 7 major Wiskott-Aldrich syndrome markets are expected to exhibit a CAGR of 4.1% during 2024-2034.

The Wiskott-Aldrich syndrome market has been comprehensively analyzed in IMARC's new report titled "Wiskott-Aldrich Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Wiskott-Aldrich syndrome (WAS) refers to a rare X-linked recessive immunodeficiency disorder that primarily affects males. It is caused by a mutation in the WAS gene, which is responsible for producing a protein called WASp. This protein plays an essential role in the development and function of specific immune cells, particularly T cells, B cells, and platelets. As a result of the gene variation, individuals suffering from the ailment may experience dysfunctional immune cells, leading to recurrent infections, eczema, and an increased risk of developing autoimmune disorders and certain cancers. Various other indications include reduced platelet counts, bleeding problems, petechiae, bruising, spontaneous nose bleeds, bloody diarrhea, etc. The diagnosis of WAS is based on a person's clinical symptoms, medical history, as well as low immunoglobulin levels. A healthcare provider may further perform DNA sequence analysis to detect pathogenic variants associated with this disease. Several other laboratory studies, such as complete blood cell count, peripheral blood smear, lymphocyte subsets, mitogen responses, etc., are also utilized to confirm a diagnosis among patients.

The increasing prevalence of genetic conditions that lead to a deficiency or malfunction of specific proteins, resulting in a weakened immune system and decreased platelet function, is primarily driving the Wiskott-Aldrich syndrome market. Furthermore, the widespread utilization of intravenous immunoglobulin to treat the illness, since it normalizes the compromised defense system and improves the quality of life in patients, is acting as another significant growth-inducing factor. Apart from this, the inflating application of stem cell transplant, which uses healthy blood-forming cells to replace unhealthy tissues, thereby reducing the severity of the disease, is also creating a positive outlook for the market. Moreover, various key players are making substantial investments in research activities to launch more potent medications that target specific immune cell receptors or signaling molecules in order to improve the immune response and alleviate symptoms. This, in turn, is further propelling the market growth. Additionally, the escalating demand for lentiviral gene therapy, in which genes can be modified, added, or deleted in order to reduce infections and bleeding episodes in patients, is expected to drive the Wiskott-Aldrich syndrome market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Wiskott-Aldrich syndrome 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 Wiskott-Aldrich syndrome 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 Wiskott-Aldrich syndrome 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 Wiskott-Aldrich syndrome market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Wiskott-Aldrich syndrome market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Wiskott-Aldrich syndrome 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

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:
Market Insights

How has the Wiskott-Aldrich syndrome 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 Wiskott-Aldrich syndrome market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the Wiskott-Aldrich syndrome 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 Wiskott-Aldrich syndrome across the seven major markets?
What is the number of prevalent cases (2018-2034) of Wiskott-Aldrich syndrome by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Wiskott-Aldrich syndrome by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with Wiskott-Aldrich syndrome across the seven major markets?
What is the size of the Wiskott-Aldrich syndrome 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 Wiskott-Aldrich syndrome?
What will be the growth rate of patients across the seven major markets?

Wiskott-Aldrich Syndrome: 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 Wiskott-Aldrich syndrome 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 Wiskott-Aldrich syndrome market?
What are the key regulatory events related to the Wiskott-Aldrich syndrome market?
What is the structure of clinical trial landscape by status related to the Wiskott-Aldrich syndrome market?
What is the structure of clinical trial landscape by phase related to the Wiskott-Aldrich syndrome market?
What is the structure of clinical trial landscape by route of administration related to the Wiskott-Aldrich syndrome 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 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Wiskott-Aldrich Syndrome - Unmet Needs

10 Wiskott-Aldrich Syndrome - Key Endpoints of Treatment

11 Wiskott-Aldrich Syndrome - Marketed Products
11.1 List of Wiskott-Aldrich Syndrome 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 Wiskott-Aldrich Syndrome - Pipeline Drugs
12.1 List of Wiskott-Aldrich Syndrome Pipeline Drugs Across the Top 7 Markets
12.1.1 Wiskott-Aldrich syndrome gene therapy - Genethon
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
12.1.2 OTL-103 - Orchard Therapeutics
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status

Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.

13. Wiskott-Aldrich Syndrome - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Wiskott-Aldrich Syndrome – 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 Wiskott-Aldrich Syndrome - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Wiskott-Aldrich Syndrome - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Wiskott-Aldrich Syndrome - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Wiskott-Aldrich Syndrome - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Wiskott-Aldrich Syndrome - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Wiskott-Aldrich Syndrome - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Wiskott-Aldrich Syndrome - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Wiskott-Aldrich Syndrome - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Wiskott-Aldrich Syndrome - 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 Wiskott-Aldrich Syndrome - Access and Reimbursement Overview

16 Wiskott-Aldrich Syndrome - Recent Events and Inputs From Key Opinion Leaders

17 Wiskott-Aldrich Syndrome Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Wiskott-Aldrich Syndrome Market – Strategic Recommendations

19 Appendix