Australia Angelman Syndrome Therapeutics Market Analysis

Australia Angelman Syndrome Therapeutics Market Analysis


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Australia During the forecast period of 2023?2030, The Angelman Syndrome Therapeutics Market is anticipated to increase from $xx Mn in 2023 to $xx Mn by 2030, recording a CAGR of xx percent. An uncommon inherited neuro-developmental condition called Angelman Syndrome is marked by a severe developmental delay, sleep issues, jerky movements, and a lot of laughter. As the illness is more common, there are more government measures to manage it. This has also helped with research and development for condition treatment. These factors collectively represent the Angelman Syndrome Therapeutics Market's driving forces. Boston Children's Hospital, the Angelman Syndrome Foundation, Rady Children's Hospital in San Diego, General Hospital Corporation, Cincinnati Children's Hospital Medical Center, and others are major competitors in the market for treating Angelman syndrome.

ID: IN10AURD005 CATEGORY: Rare Diseases GEOGRAPHY: Australia AUTHOR: Gaurav Bhongale

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Australia Angelman Syndrome Therapeutics Market Analysis Summary

Australia Angelman Syndrome Therapeutics Market is valued at around $2.56 Mn in 2022 and is projected to reach $5.73 Mn by 2030, exhibiting a CAGR of 10.6% during the forecast period 2023-2030.

Angelman syndrome is a rare and complicated hereditary disorder that mostly affects the nervous system. Intellectual disability, issues with balance and movement, delayed development, and significant speech impairment are some of this health condition's most prevalent defining symptoms. Recurrent seizures and microcephaly, or small heads, can both occur in a number of affected children. AS is typically diagnosed between the ages of 3 and 7. The Angelman syndrome is expected to afflict more than 1 in 12 to 20 thousand people. There are numerous genetic alterations that cause AS, but they all result in a similar clinical presentation. A loss in the maternally contributed portion of chromosome 15q11–q13 accounts for about 70% of cases. DNA is methylation in the AS crucial region.

Typically, an AS diagnosis and test are performed after the infant is born. The majority of people with AS may be found via a blood test, which determines whether or not the UBE3A gene is operating normally. To identify the syndrome, fluorescent in situ hybridization (FISH) and CGH Array tests are used.

A recent study that disclosed new information about the disease process for Angelman syndrome (AS) found increases in autophagy, a method for removing cellular trash. These findings show that UBE3A deficiency enhances autophagy activity. One of the areas of the brain most affected by AS is the cerebellum. UX111, DTX401, DTX301, and UX701 are just a few of the gene therapy candidates in Ultragenyx's pipeline that have the potential to revolutionize the way many rare genetic illnesses are treated. Recently, the FDA authorized modifications to the GTX-102 Phase 1/2 trial in young patients with Angelman syndrome, enabling Ultragenyx to coordinate dosage scopes and speed up enrolment in the United States.

Boston Children's Hospital, the Angelman Syndrome Foundation, Rady Children's Hospital in San Diego, General Hospital Corporation, Cincinnati Children's Hospital Medical Center, and others are major competitors in the market for treating Angelman syndrome.

Market Dynamics

MarketDrivers

  • Rising research and development spending

In the upcoming years, it is projected that the global market for treating Angelman syndrome would grow favourably as research and development spending increases to find better and more efficient treatments.

  • Governmental programs

Rising government initiatives from nations across the world in an effort to create new and more effective treatment methods for Angelman syndrome are also projected to support the growth of the global market for treatments for Angelman syndrome in the upcoming years. The Global Angelman Syndrome Registry is an online database run by patient organizations that aim to compile data on the natural history of people with Angelman Syndrome in both children and adults.

  • Strengthening Production and Development

Increasing research and development spending has led to the introduction of new technologies and the invention of products in the worldwide market for the treatment of Angelman syndrome.

  • Creation of novel and specific

Researchers are currently working to make the treatment process more convenient by using effective treatment and technology.

 

Market Development

The prevalence of the syndrome has considerably increased around the globe. The first biobank in Australia with the aim of promoting research and treatment of rare genetic diseases caused by changes in genes on chromosome 15—including Prader-Willi Syndrome (PWS) and Angelman Syndrome (AS)—will be created at the Murdoch Children's Research Institute (MCRI).

RARE revealed that the FDA had authorized a protocol amendment after examining the phase I/II research of GTX-102 in young individuals with the uncommon neurogenetic disorder Angelman syndrome. The decision of this regulatory authority will let Ultragenyx to align dose levels in the United States with those being utilized in research cohorts outside of the United States.

"We have 500,000 children estimated to have Angelman syndrome in the globe, and we've identified under 10,000," claims Allyson Berent, chief science officer at the Foundation for Angelman Syndrome Therapeutics (FAST). We've started a number of newborn screening initiatives and a number of global diagnostic efforts for people who are not connected, underdiagnosed, or misdiagnosed in order to help fix that. In spite of the fact that there are currently no approved treatments for the uncommon neurogenetic disorder known as Angelman syndrome, Berent claims that his organization is advancing 13 potential treatments from mice to people, with a number of clinical trials already underway or scheduled to begin in the next 18 to 24 months.

Market Restraints

Children with Angelman syndrome may develop a wide range of neurological symptoms, all of varying degrees of severity. Ataxia, or the inability to coordinate voluntary movements, seizures, trouble sleeping, problems with movement and balance, a limited or impossible ability to converse, and others may also be present. Lack of understanding of this disease not only results in inaccurate diagnosis but also has an effect on the expansion of the global therapeutic market. The market for treatments for Angelman syndrome is also constrained by a paucity of data on the illness's prevalence and financial burden of care.

Key players

uniQure Ionis Pharmaceuticals Alnylam Pharmaceuticals Catalyst Biosciences Pfizer Sanofi Shire plc BioMarin Pharmaceutical Inc. Orchard Therapeutics plc PTC Therapeutics Inc.

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Market Segmentations For Australia Angelman Syndrome Therapeutics Market

By Diagnosis

  • Neonatal detection of the disorder
  • Blood test for UBE3A functionality
  • Fluorescent in situ hybridization (FISH) test
  • CGF Array Test

By Treatment

  • Physical Therapy
  • Communication Therapy
  • Behaviour Therapy
  • Others

By dosage form

  • Oral
  • Injectables
  • Others

By Service Provider

  • Hospitals
  • Specialty Clinics
  • Surgical Centers
  • Others

Insights10 will provide you with the reports within 10 key parameters which are:

  1. Market Overview
  2. Market Growth Drivers & Restraints
  3. Epidemiology of Disease Type
  4. Market Segmentation
  5. Market Share
  6. Competitive Landscape
  7. Key Company Profiles
  8. Healthcare Policies & Regulatory Framework
  9. Reimbursement Scenario
  10. Factors Driving Future Growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision-making for business stakeholders. Our focused approach to developing reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

 

Stage I: Market Data Collection

Primary Interviews: We have developed a network of experts, freelancers, and researchers across countries through which we engage with local experts to gather key data points and assumptions about each market. We also engage regularly with some of the best market research agencies such as Atheneum, GuidePoint, GLG, etc. to conduct surveys and interviews, and build intelligence. We have language translators as a part of our team, who between them can cover 30+ languages allowing us to extract better local insights.

Secondary Data Collection: We have developed strong expertise and experience in secondary data collection methods for developing unique data sets and research material. We gather data from multiple reliable sources to maintain a high level of accuracy and consistency. The market data is analyzed and forecasted using appropriate statistical and coherent models. The report offers an overall analysis of the market size, growth, and market share as well as a segment-level analysis of the specific market. Our report includes precise, to-the-point information related to the overall market, competition, growth drivers, challenges, regulatory updates, and competition.

Data Sources: We have access to multiple highly reliable free and subscription data sources. We have many years of experience to understand which sources are more dependable for what and which to prefer for the reliable and latest information. The key sources of information include the following, but are not limited to:

 

 

Stage II: Market Data Analysis and Statistical Model

Market Trends: We generally look at macro parameters and micro indicators. The macro parameters include changes in government policies, demand and supply of the market, government intervention programs, and major market share. The micro indicators are GDP growth, market size, market volume, etc. We also understand nuances specific to each country like the US, Canada, India, Germany, etc., and have worked across 60+ countries and hence not only understand global trends but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. starting with the large overall market and segmenting different areas and the bottom-up approach i.e. starting with population and epidemiology and rolling up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

The top-down approach is mainly used for new product forecasting and the bottom-up approach is used for demand estimation of any product for different countries summed up to form the total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as Asia-Pacific, Africa, Europe, Middle East, North America, and  Latin America for the period of 2022 to 2030. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape, and policy and regulatory scenario, and the quantitative analysis covers different market estimates and forecasts.

Data Triangulation & Validation:

Data triangulation of various sources and results of the research are carried out by benchmarking with reliable sources such as industry statistics, statistical databases, and company-level averages, etc.

We make sure to finalize the numbers in alignment with the market research. Firstly, our internal experts ensure thorough validation and checking to ensure accurate and precise analysis and then validation is also done using a multiple-data analysis model. Two-level validation is done and entails the finalization of the report scope and the way of representation pattern.

 

 

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analyzed and synthesized. The second series of interviews are done if necessary to check and validate. The future opportunities are analyzed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results is then, interpreted by considering the following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing the regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue/volume
  • ​Analyzing current needs and determining penetration to estimate the market

Insights: Our reports deliver actionable insights backed with supporting facts and figures to assist you in achieving exemplary growth. Our in-depth analyses are interspersed with relevant insights and statistics to offer an executive-level view of a given market. The description helps in correlating many minor factors affecting the market and their impact on the different segments within the market.

Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

Presentation & Reporting: The market research report is presented in different forms such as charts by using a scientific approach for easy understanding. Historic, current, and future analysis is provided for each market in terms of both value and volume. The size of the market is interpreted in the US Dollar value and the respective unit, based on the product, for volume consumption.

The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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  • Intelligent insights to take informed business decisions
  • Qualitative, acute and result oriented market analysis
  • Market size and forecasts from 2022 to 2030
  • Opportunities for expansion and in-depth market analysis
  • Segmentation and regional revenue forecasts
  • Analysis of the market share and competitive landscape
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Last updated on: 04 July 2023
Updated by: Riya Doshi

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