Angelman Syndrome Market

Key Highlights

• As per DelveInsight, the Angelman Syndrome Market is expected to expand at a healthy growth rate during the forecast period (2025-2034), owing to the launch of new therapies in the market and the rise in the number of cases. 
• Several major pharma and biotech companies such as GeneTx Biotherapeutics, GEXVal, Ionis Pharmaceuticals, Neuren Pharmaceuticals, Ovid Therapeutics, PTC Therapeutics, Roche, Sarepta Therapeutics, StrideBio, Taysha Gene Therapies, Ultragenyx Pharmaceutical, among others, are actively working in the Angelman Syndrome Market.

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DelveInsight's "Angelman Syndrome Market Insights, Epidemiology, and Market Forecast-2034" report delivers an in-depth understanding of the Angelman Syndrome, historical and forecasted epidemiology as well as the Angelman Syndrome market trends in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom) and Japan.

The Angelman Syndrome market report provides current treatment practices, emerging drugs, Angelman Syndrome market share of the individual therapies, and current and forecasted Angelman Syndrome market size from 2020 to 2034 segmented by seven major markets. The Report also covers current Angelman Syndrome treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the Angelman Syndrome market.

Geography Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2020-2034

Angelman Syndrome: Disease Understanding and Treatment Algorithm

According to the National Organization for Rare Disorders, Angelman syndrome is a rare genetic and neurological disorder characterized by severe developmental delay and learning disabilities; absence or near absence of speech; inability to coordinate voluntary movements (ataxia); tremulousness with jerky movements of the arms and legs and a distinct behavioral pattern characterized by a happy disposition and unprovoked episodes of laughter and smiling.

Angelman Syndrome is typically caused by a deletion or mutation of the UBE3A gene on chromosome 15, which leads to a lack of functional UBE3A protein in the brain. Individuals diagnosed with Angelman syndrome typically exhibit developmental challenges that become evident within the first 6 to 12 months of life. As they progress into early childhood, a constellation of common signs and symptoms further characterizes this neurogenetic disorder. These manifestations often encompass difficulties with walking and balance, gastrointestinal complications, seizures, and limited or absent speech. It 

Moreover, individuals with Angelman syndrome may experience sleep disturbances, hyperactivity, and a fascination with water. Their distinctive behaviors, such as frequent laughter and a cheerful demeanor, can provide valuable diagnostic clues. It's important to note that these symptoms can vary in severity among affected individuals, and a comprehensive assessment by healthcare professionals is crucial for a precise diagnosis and tailored care. Furthermore, ongoing research and support organizations continue to contribute to our understanding of Angelman syndrome and the development of interventions to improve the quality of life for those affected.

A diagnosis of Angelman syndrome can typically be established through a comprehensive patient history, a meticulous clinical assessment, and the identification of distinctive clinical markers. Approximately 80% of cases can be verified using specialized blood tests, including DNA methylation analysis. Moreover, the characteristic deletion of chromosome 15q11-q13 in the body's cells, a hallmark of the syndrome, can be most commonly identified through techniques such as Fluorescent In Situ Hybridization (FISH) or microarray chromosome analysis.

Angelman Syndrome Epidemiology 

The Angelman Syndrome epidemiology section provides insights about the historical and current Angelman Syndrome patient pool and forecasted trends for individual seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Angelman Syndrome market report also provides the diagnosed patient pool and their trends along with assumptions undertaken. 

The disease epidemiology covered in the report provides historical as well as forecasted Angelman Syndrome epidemiology scenario in the 7MM covering the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan from 2020 to 2034.

Key Findings

• According to the National Organization for Rare Disorders (2022), Angelman syndrome affects males and females in equal numbers. The prevalence of Angelman syndrome is estimated to be approximately 1 in 12,000-20,000 people in the general population.

• As per Angelman UK (2022), Angelman Syndrome is a rare neurological disorder affecting around 1:20,000 births. Characteristic features include delayed development, severe learning difficulties, little or no speech, and issues with movement and balance.

Country Wise- Angelman Syndrome Epidemiology

The epidemiology segment also provides the Angelman Syndrome epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

The Report Covers the Angelman Syndrome Epidemiology Segmented as

• Total Prevalent cases of Angelman Syndrome
• Diagnosed Prevalent Cases of Angelman Syndrome
• Angelman Syndrome Associated with Clinical Manifestations 
• Treated Cases of Angelman Syndrome

Get detailed insights into the historical as well as forecasted epidemiology trends in the 7MM, at: Angelman Syndrome Epidemiology Forecast

Angelman Syndrome Recent Developments

• In September 2025, MavriX Bio announced that the U.S. FDA granted Fast Track designation to MVX-220, its investigational AAV gene therapy for Angelman syndrome (AS), aiming to accelerate development of this potential treatment for the rare neurogenetic disorder.
• In May 2025, MavriX Bio announced FDA clearance of its IND for MVX-220, an AAV gene therapy targeting Angelman syndrome (AS). The upcoming ASCEND-AS first-in-human trial will assess the safety and efficacy of MVX-220, designed to restore UBE3A gene function, in both adult and pediatric patients with various AS genotypes.

Angelman Syndrome Drug Chapters

The drug chapter segment of the Angelman Syndrome report encloses the detailed analysis of Angelman Syndrome marketed drugs and late-stage (Phase-III and Phase-II) Angelman Syndrome pipeline drugs. It also helps to understand the Angelman Syndrome clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Angelman Syndrome Marketed Drugs

The report provides the details of the marketed products/off-label treatments available for Angelman Syndrome treatment.

Angelman Syndrome Emerging Drugs

The report provides the details of the emerging therapies under the late and mid-stage of development for Angelman Syndrome treatment.

Angelman Syndrome Market Outlook

The Angelman Syndrome market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Angelman Syndrome market trends by analyzing the impact of current Angelman Syndrome therapies on the market, unmet needs, drivers and barriers, and demand for better technology.

This segment gives a thorough detail of Angelman Syndrome market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Angelman Syndrome market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, the Angelman Syndrome market in 7MM is expected to witness a major change in the study period 2020-2034.

Key Findings

This section includes a glimpse of the Angelman Syndrome market in 7MM.

The United States Market Outlook

This section provides the total Angelman Syndrome market size and market size by therapies in the United States.

EU-5 Countries: Market Outlook

The total Angelman Syndrome market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

Japan Market Outlook

The total Angelman Syndrome market size and market size by therapies in Japan are also mentioned.

Angelman Syndrome Drugs Uptake

This section focuses on the rate of uptake of the potential Angelman Syndrome drugs recently launched in the Angelman Syndrome market or expected to get launched in the market during the study period 2020-2034. The analysis covers Angelman Syndrome market uptake by drugs; patient uptake by therapies; and sales of each drug.   

Angelman Syndrome Drugs Uptake helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allow the comparison of the drugs on the basis of Angelman Syndrome market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Angelman Syndrome Pipeline Development Activities

The Angelman Syndrome report provides insights into different therapeutic candidates in Phase II, and Phase III stage. It also analyses Angelman Syndrome companies involved in developing targeted therapeutics.

Pipeline Development Activities

The Angelman Syndrome report covers the detailed information of collaborations, acquisition, and merger, licensing, patent details, and other information for Angelman Syndrome emerging therapies.

Explore more about the emerging therapies and key companies actively working in the market: Angelman Syndrome Pipeline Insights

Angelman Syndrome Market Assessment and Key Companies in the Domain

DelveInsight's expert analysts anticipate a promising trajectory for the market, driven primarily by several key factors. Notably, the market is poised for positive growth owing to the rising prevalence of the condition, enhanced diagnostic capabilities, and the eagerly awaited introduction of innovative therapeutic options in the near future.

The escalating incidence of the targeted condition underscores the urgent need for advanced treatments. Furthermore, improved diagnostic methods are expected to lead to early and accurate detection, allowing for timely intervention and better patient outcomes.

In addition to these factors, the market's favorable outlook is bolstered by the anticipated arrival of novel therapies. These innovative treatments have the potential to revolutionize patient care by offering improved efficacy, reduced side effects, and enhanced overall quality of life. As the healthcare landscape continues to evolve, it is essential to stay updated on these advancements to provide the best possible care and support for those affected by the condition.

The market has a promising outlook with some of the best potential emerging therapies. Some of the drugs in the pipeline include GTX-102 (GeneTx Biotherapeutics/Ultragenyx Pharmaceutical), ION582 (Ionis Pharmaceuticals/Biogen/ Emmes Company), NNZ-2591 (Neuren Pharmaceuticals), among others.

Reimbursement Scenario in Angelman Syndrome Market

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In a report, we take reimbursement into consideration to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

KOL- Views

To keep up with current Angelman Syndrome market trends, we take KOLs and SMEs ' opinion working in the Angelman Syndrome domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Angelman Syndrome market trends. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the Angelman Syndrome Market by using various Competitive Intelligence tools that include - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of Angelman Syndrome, explaining its causes, signs and symptoms, pathophysiology, diagnosis and currently available therapies
• Comprehensive insight has been provided into the Angelman Syndrome epidemiology and treatment in the 7MM
• Additionally, an all-inclusive account of both the current and emerging therapies for Angelman Syndrome is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
• A detailed review of the Angelman Syndrome market; historical and forecasted is included in the report, covering drug outreach in the 7MM
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Angelman Syndrome market

Report Highlights

• In the coming years, the Angelman Syndrome market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
• The companies and academics are working to assess challenges and seek opportunities that could influence Angelman Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition      
• Several major Angelman Syndrome companies are involved in developing therapies to improve the treatment outlook. The launch of emerging therapies will significantly impact the Angelman Syndrome market
• A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Angelman Syndrome
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities

Angelman Syndrome Market Report Insights

• Angelman Syndrome Patient Population
• Angelman Syndrome Therapeutic Approaches
• Angelman Syndrome Pipeline Analysis
• Angelman Syndrome Market Size and Trends
• Angelman Syndrome Market Opportunities
• Impact of Upcoming Angelman Syndrome Therapies

Angelman Syndrome Market Report Key Strengths

• 10 Years Forecast
• 7MM Coverage
• Angelman Syndrome Epidemiology Segmentation
• Key Cross Competition in the Angelman Syndrome Market
• Highly Analyzed Angelman Syndrome Market
• Angelman Syndrome Drugs Uptake

Angelman Syndrome Market Report Assessment

• Current Angelman Syndrome Treatment Practices
• Unmet Needs in the Angelman Syndrome Market
• Angelman Syndrome Pipeline Product Profiles
• Angelman Syndrome Market Attractiveness
• Angelman Syndrome Market Drivers and Barriers

Key Questions

Market Insights:

• What was the Angelman Syndrome drug class share (%) distribution in 2020 and how it would look like in 2034?
• What would be the Angelman Syndrome total market size as well as market size by therapies across the 7MM during the forecast period (2020-2034)?
• What are the key findings pertaining to the market across 7MM and which country will have the largest Angelman Syndrome market size during the forecast period (2020-2034)?
• At what CAGR, the Angelman Syndrome market is expected to grow by 7MM during the forecast period (2020-2034)?
• What would be the Angelman Syndrome market outlook across the 7MM during the forecast period (2020-2034)?
• What would be the Angelman Syndrome market growth till 2034, and what will be the resultant market Size in the year 2034?
• How would the unmet needs affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

• What are the disease risk, burden, and regional/ethnic differences of the Angelman Syndrome?
• What are the key factors driving the epidemiology trend for seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What is the historical Angelman Syndrome patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What would be the forecasted patient pool of Angelman Syndrome in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• Where will be the growth opportunities in the 7MM with respect to the patient population pertaining to Angelman Syndrome?
• Out of all 7MM countries, which country would have the highest prevalent population of Angelman Syndrome during the forecast period (2020-2034)?
• At what CAGR the patient population is expected to grow in 7MM during the forecast period (2020-2034)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

• What are the current options for the Angelman Syndrome treatment in addition to the approved therapies?
• What are the current treatment guidelines for the treatment of Angelman Syndrome in the USA, Europe, and Japan?
• What are the Angelman Syndrome marketed drugs and their respective MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
• How many companies are developing therapies for the treatment of Angelman Syndrome?
• How many therapies are in-development by each company for Angelman Syndrome treatment?
• How many are emerging therapies in mid-stage, and late stage of development for Angelman Syndrome treatment?
• What are the key collaborations (Industry - Industry, Industry - Academia), Mergers and acquisitions, licensing activities related to the Angelman Syndrome therapies?
• What are the recent novel therapies, targets, mechanisms of action and technologies being developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Angelman Syndrome and their status?
• What are the current challenges faced in drug development?
• What are the key designations that have been granted for the emerging therapies for Angelman Syndrome?
• What are the global historical and forecasted market of Angelman Syndrome?

Reasons to buy

• The report will help in developing business strategies by understanding trends shaping and driving the Angelman Syndrome market
• To understand the future market competition in the Angelman Syndrome market and Insightful review of the key market drivers and barriers
• Organize sales and marketing efforts by identifying the best opportunities for Angelman Syndrome in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan
• Identification of strong upcoming Angelman Syndrome companies in the market will help in devising strategies that will help in getting ahead of competitors
• Organize sales and marketing efforts by identifying the best opportunities for Angelman Syndrome market
• To understand the future market competition in the Angelman Syndrome market