Angelman Syndrome (AS) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Angelman Syndrome (AS) Market Outlook

Thelansis’s “Angelman Syndrome (AS) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" 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 (AS) is a rare genetic and neurological disorder characterized by severe developmental delays and learning disabilities. Individuals with AS exhibit an inability to coordinate voluntary movements (ataxia), a near absence of speech, and tremulousness with jerky arm and leg movements. They also exhibit a distinct behavioral pattern characterized by a happy disposition, unprovoked episodes of laughter and smiling, and frequent hand-flapping movements. Children diagnosed with Angelman syndrome typically display a cheerful and excitable demeanor, often accompanied by frequent smiling and laughter. They also commonly show signs of hyperactivity, have a short attention span, and a fascination with water. Additionally, many affected children experience difficulty sleeping and require less sleep than usual. It's important to note that Angelman syndrome does not impact stature, limb size, or sexual development. Puberty and fertility are not affected, with menstruation and spermatogenesis occurring at a similar time as in other children. As individuals with Angelman syndrome age, they tend to become less excitable, and their sleeping difficulties often improve. However, they continue to grapple with intellectual disability, severe speech impairment, and a susceptibility to seizures throughout their lives. In adulthood, individuals with Angelman syndrome exhibit distinct facial features described as "coarse." Other common characteristics include unusually fair skin with light-colored hair and an abnormal side-to-side curvature of the spine, known as scoliosis.

• The prevalence of Angelman syndrome ranges from 1 to 1.5 cases per 40,000 population in the USA.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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