엔젤만 증후군(AS) : 시장 전망, 역학, 경쟁 구도 시장 예측 리포트(2025-2035년)

Angelman Syndrome (AS) Market Outlook

Thelansis's "Angelman Syndrome (AS) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report - 2025 To 2035" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Angelman Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Angelman Syndrome (AS) Overview

Angelman Syndrome is a neurogenetic disorder caused by loss of maternal UBE3A expression, impairing ubiquitin-mediated protein degradation and synaptic function. It presents with severe intellectual disability, absent speech, ataxic gait, seizures, and a characteristically happy demeanor. Diagnosis combines methylation analysis, UBE3A sequencing, and chromosomal microarray; seizure burden and functional impairment reflect severity. Seizure management incorporates valproate, levetiracetam, and cannabidiol, the latter offering efficacy in refractory myoclonic episodes with limited cognitive sedation. ASO therapies targeting UBE3A antisense transcripts to restore paternal gene expression are in advanced clinical trials. Regular neurodevelopmental monitoring, augmentative communication, and family-centred support underpin lifelong quality-of-life management.

Key Highlights

• In Germany, total prevalent Angelman syndrome cases are projected to increase from approximately 6,772 in 2025 to 6,896 by 2035.
• Angelman syndrome remains a rare genetic neurodevelopmental disorder associated with severe cognitive and motor impairment.
• Patients experience developmental delays, speech impairment, seizures, and behavioral abnormalities.
• Significant unmet need persists due to limited disease-modifying treatment options.
• Emerging gene therapies and targeted neurological treatments are transforming the future treatment landscape.

Market Overview

• The Italy Angelman syndrome market is projected to grow from approximately $28 MN to $195 MN by 2035.
• Market growth is supported by:
• Increasing development of gene-targeted therapies
• Improved diagnosis and patient identification
• Market expansion is expected to be driven by high-value rare disease treatments.
• Future growth will depend on successful commercialization of disease-modifying and gene replacement therapies.

Insights driven by robust research, including:

• In-depth interviews with leading KOLs and payers
• Physician surveys
• RWE analysis for claims and EHR datasets
• Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

• Detailed Report (PDF)
• Market Forecast Model (MS Excel-based automated dashboard)
• Epidemiology (MS Excel; interactive tool)
• Executive Insights (PowerPoint presentation)
• Others: regular updates, customizations, consultant support
• As per Thelansis's policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

• 10-year market forecast (2025-2035)
• Bottom-up patient-based market forecasts validated through the top-down sales methodology
• Covers clinically and commercially-relevant patient populations/ line of therapies
• Annualized drug-level sales and patient share projections
• Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
• Detailed methodology/sources & assumptions
• Graphical and tabular outputs
• Users can customize the model based on requirements

Key business questions answered:

• How can drug development and lifecycle management strategies be optimized across G8 markets (US, EU5, Japan, and China)?
• How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
• What is the 10-year market outlook for sales and patient share?
• Which events will have the greatest impact on the market's trajectory?
• What insights do interviewed experts provide on current and emerging treatments?
• Which pipeline products show the most promise, and what is their potential for launch and future positioning?
• What are the key unmet needs and KOL expectations for target profiles?
• What key regulatory and payer requirements must be met to secure drug approval and favorable market access?

Countries Covered

• G8
  • United States
  • EU5
    • France
    • Germany
    • Italy
    • Spain
    • U.K.
  • Japan
  • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

Companies Mentioned

• Ionis Pharmaceuticals, Inc.
• Ultragenyx Pharmaceutical Inc.
• MavriX Bio, LLC
• Neuren Pharmaceuticals Limited
• Hoffmann-La Roche
• Healx AI

Table of Contents

1. Key Findings and Analyst Commentary

• Key trends: market snapshots, SWOT analysis, commercial benefits and risks, etc.

2. Disease Context

• Disease definition, classification, etiology and pathophysiology, drug targets, etc.

3. Epidemiology

• Key takeaways
• Incidence / Prevalence
• Diagnosed and Drug-Treated populations
• Comorbidities
• Other relevant patient segments

4. Market Size and Forecast

• Key takeaways
• Market drivers and constraints
• Drug-class specific trends
• Country-specific trends

5. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Notable late-phase emerging therapies - profiles, launch expectations, KOL insights
• Notable early-phase pipeline

6. Unmet Need and TPP Analysis

• Top unmet needs and future attainment by emerging therapies
• TPP analysis and KOL expectations

7. Regulatory and Reimbursement Environments (by country and payer insights)

8. Appendix (e.g., bibliography, methodology)