Angelman Syndrome (AS) is a complex genetic disorder that primarily affects the nervous system. The characteristic features of this condition include delayed development, intellectual disability, severe speech impairment, and problems with movement and balance (ataxia).
Angelman Syndrome Epidemiology
- Total prevalent cases of Angelman Syndrome in 7 MM in 2017 was 57,716
- The prevalence of Angelman Syndrome in 2017
- United States – 21,715
- Germany – 6,448
- Spain – 3,917
Angelman Syndrome Epidemiology Segmentation in 7MM from 2017 to 2030
- Total Prevalent cases of Angelman Syndrome
- Diagnosed Prevalent Cases of Angelman Syndrome
- Angelman Syndrome Cases by Mutation Types
- Angelman Syndrome associated with Clinical Manifestations
Angelman Syndrome Market
The therapeutic Angelman Syndrome market in 7MM in 2017 was USD 255 Million
Angelman Syndrome Market Drivers
- Biomarker Studies
- Increasing Prevalence of Angelman Syndrome
- Molecular Level Diagnosis
Angelman Syndrome Market Barriers
- Lack of awareness
- Lack of late stage clinical trials
- Lack of Data
- Economic Burden
Angelman Syndrome Emerging Drugs
- Gaboxadol: Ovid Therapeutics
- GTX-101: GeneTx Biotherapeutics
- GT-AS/AGIL-AS: PTC Therapeutics
1 Key Insights
2 Executive Summary of Angelman Syndrome
3 SWOT Analysis for Angelman Syndrome
4 Angelman Syndrome Market Overview at a Glance
4.1 Market Share (%) Distribution of Angelman Syndrome in 2017
4.2 Market Share (%) Distribution of Angelman Syndrome in 2030
5 Disease Background and Overview
5.1 Introduction
5.2 Cause
5.3 Signs and Symptoms
5.4 Related Disorders and Differential Diagnosis
5.5 Complications
5.6 Genetics of Angelman syndrome
5.7 Molecular Genetic Pathogenesis
5.8 Diagnosis
5.8.1 Clinical criteria for diagnosis
5.8.2 Disease History and Medical Conditions
5.9 Diagnostic Algorithm
6 Epidemiology and Patient Population
6.1 Key Findings
6.2 7MM Total Prevalent Patient Population of Angelman syndrome
7 Country Wise-Epidemiology of Angelman syndrome
7.1 The United States
7.1.1 Assumptions and Rationale
7.1.2 Total Prevalent Cases of Angelman Syndrome in the United States
7.1.3 Diagnosed Prevalent Cases of Angelman Syndrome in the United States
7.1.4 Angelman Syndrome cases by Mutation Types in the United States
7.1.5 Angelman Syndrome associated with Clinical Manifestations in the United States
7.2 EU5
7.2.1 Assumptions and Rationale
7.3 Germany
7.3.1 Total Prevalent Cases of Angelman Syndrome in Germany
7.3.2 Diagnosed Prevalent Cases of Angelman Syndrome in Germany
7.3.3 Angelman Syndrome cases by Mutation Types in Germany
7.3.4 Angelman Syndrome associated with Clinical Manifestations in Germany
7.4 France
7.4.1 Total Prevalent Cases of Angelman Syndrome in France
7.4.2 Diagnosed Prevalent Cases of Angelman Syndrome in France
7.4.3 Angelman Syndrome cases by Mutation Types in France
7.4.4 Angelman Syndrome associated with Clinical Manifestations in France
7.5 Italy
7.5.1 Total Prevalent Cases of Angelman Syndrome in Italy
7.5.2 Diagnosed Prevalent Cases of Angelman Syndrome in Italy
7.5.3 Angelman Syndrome cases by Mutation Types in Italy
7.5.4 Angelman Syndrome associated with Clinical Manifestations in Italy
7.6 Spain
7.6.1 Total Prevalent Cases of Angelman Syndrome in Spain
7.6.2 Diagnosed Prevalent Cases of Angelman Syndrome in Spain
7.6.3 Angelman Syndrome cases by Mutation Types in Spain
7.6.4 Angelman Syndrome associated with Clinical Manifestations in Spain
7.7 The United Kingdom
7.7.1 Total Prevalent Cases of Angelman Syndrome in the United Kingdom
7.7.2 Diagnosed Prevalent Cases of Angelman Syndrome in the United Kingdom
7.7.3 Angelman Syndrome cases by Mutation Types in the UK
7.7.4 Angelman Syndrome associated with Clinical Manifestations in the UK
7.8 Japan
7.8.1 Assumptions and Rationale
7.8.2 Total Prevalent Cases of Angelman Syndrome in Japan
7.8.3 Diagnosed Prevalent Cases of Angelman Syndrome in Japan
7.8.4 Angelman Syndrome cases by Mutation Types in Japan
7.8.5 Angelman Syndrome associated with Clinical Manifestations in Japan
8 Treatment and Management of Angelman syndrome
8.1 Recommendations for the management of Angelman Syndrome
8.2 Future treatment options for Angelman Syndrome
8.2.1 Gene Therapy
8.2.2 Topoisomerase Inhibitors
8.2.3 Minocycline
8.2.4 Cannabidiol (CBD)
8.2.5 Protein Phosphatase 2A (PP2A) Inhibitor
9 Unmet Needs
10 Organizations contributing toward Angelman Syndrome
11 KOL’s Views: Angelman Syndrome
12 Patient Journey
12.1 Journey toward a Diagnosis: Diagnosing a Rare form of Angelman Syndrome
13 Case Reports
13.1 A Case of Fundus Oculi Albinoticus Diagnosed as Angelman Syndrome by Genetic Testing – A Japanese Case Study
13.2 Two Angelman families with unusually advanced neurodevelopment carry a start codon variant in the most highly expressed UBE3A isoform: a case report of US
13.3 Angelman Syndrome: A Case Series Assessing Neurological Issues in Adulthood – A European Case Report
14 Emerging Therapies
14.1 Gaboxadol: Ovid Therapeutics
14.1.1 Product Description
14.1.2 Other Development Activities
14.1.3 Clinical Development
14.1.4 Safety and Efficacy
14.2 GeneTx Biotherapeutics
14.2.1 GTX-102
15 Future Gene Therapies
15.1 GeneTx Biotherapeutics
15.1.1 GTX-101
15.2 PTC Therapeutics: GT-AS/AGIL-AS:
15.2.1 Product Description
15.2.2 Other Development Activities
15.3 Sarepta Therapeutics and StrideBio
15.3.1 Development Activities
16 Angelman syndrome: Seven Major Market Analysis
16.1 Key Findings
16.2 Market Size of Angelman Syndrome in 7MM
17 Market Outlook by Country
17.1 The United States: Market Outlook
17.1.1 United States Market Size
17.2 EU-5 Countries: Market Outlook
17.2.1 Germany
17.2.2 France
17.2.3 Italy
17.2.4 Spain
17.2.5 United Kingdom
17.3 Japan: Market Outlook
17.3.1 Total Market Size of Angelman syndrome
17.3.2 Market Size of Angelman Syndrome by Therapies
18 Market Drivers
19 Market Barriers
20 Market Access and Reimbursement
21 Appendix
21.1 Report Methodology
22 DelveInsight Capabilities
23 Disclaimer
24 About DelveInsight
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