UK Rett Syndrome Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2610

  • Format: PPT, PDF

UK Rett Syndrome Therapeutics Market: Segmented by Diagnosis, Treatment, and Distribution Channels – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021


SKU: 2610 Categories: ,

Report Overview

The UK Rett Syndrome Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Rett syndrome is a rare genetic neurological disorder that is more common among girls and leads to severe impairment affecting their ability to speak, walk and eat. Rett syndrome is recognized in children between 6-18 months caused by mutations on the X-chromosome over a gene called MECP2. Rett syndrome can cause a wide range of disabilities from mild to severe. Common symptoms of Rett syndrome include slowed growth, unusual eye movements, agitation and irritability, problems with hand movements, poor language skills, muscles and coordination problems, and trouble with breathing.

Health care professionals diagnosed Rett syndrome on the basis of MECP2 mutation. It is a rare condition so doctors also diagnosed other conditions such as prenatal brain disorder, autism spectrum disorder, metabolic disorder, and cerebral palsy to confirm Rett syndrome.

As per one of the studies conducted, more than 1500 people with Rett syndrome have been diagnosed in the UK. Moreover, the prevalence of Rett syndrome in females is around 1;10,000 while the prevalence of Rett syndrome in males is around 1:40,000.

Market Growth Drivers

The rise in the female population, expenditure on public healthcare programs and rising healthcare spending, availability of development of new drugs, and use of advanced technologies drive the Rett syndrome market growth. Increasing incidence and prevalence of Rett syndrome also anticipated enhancing the market growth.

However, increased focus on gene therapy treatment also boosts up the Rett syndrome market growth. It is expected to account for the largest market share over the forecast period for the Rett syndrome treatment market due to better healthcare infrastructure, rising economic growth, and an increase in the private healthcare sector.

Market Restraints

However, the high cost of treatment and rising R&D expenditure for the treatment of Rett syndrome may hamper the Rett syndrome market growth. Under-enrolment of participants in trials is one of the biggest challenges for researchers and trial sponsors, and it can slow the development of a potential treatment.


1. UK Rett Syndrome Therapeutics Market Overview………………………
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Rise in Female Population & Increasing Incidence of Rett Syndrome
B. Increased Focus on Gene Therapy Treatment
C. Rising Economic Growth & Healthcare Spending
D. Huge Financial support to the researchers for developing novel interventions
Market Restraints
A. Higher Cost of Treatment and Rising R&D Expenditure
B. Under Enrolment of Participants in Trials
3. Major Types of Rett Syndrome………………………………………….
A. Classical Rett Syndrome
B. Atypical Rett Syndrome
4. Rett Syndrome Therapeutics Market Segmentation…………………..
A. By Diagnosis
I. Polysomnography
II. Electroencephalography (EEG)
III. Electromyography (EMG)
IV. Plethysmography bands
V. Electrocardiography (ECG)
VI. Airflow probes
VII. Pulse oximetry
VIII. Genetic Blood Test
IX. Others
B. By Treatment
I. Physical Therapy
II. Occupational Therapy
III. Speech-Language Therapy
IV. Behavioural Therapy
V. Nutritional Therapy
VI. Physiotherapy
VII. Antileptic Drugs
VIII. Beta Blockers
IX. Others
C. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. Rett Syndrome Major Drugs Market Share………………………………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………….
A. Newron Company overview, Product & Services, Strategies & Financials
B. GW Research Company overview, Product & Services, Strategies & Financials
C. Edison Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape……………………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………………
A. New Trends and Development of Rett Syndrome Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Diagnosis:

On the basis of diagnosis, the Rett syndrome therapeutics market can be classified into

  • Polysomnography
  • Electroencephalography (EEG)
  • Electromyography (EMG)
  • Plethysmography bands
  • Electrocardiography (ECG)
  • Airflow probes
  • Pulse oximetry
  • Genetic Blood Test
  • Others

Polysomnography (PSG) is a study or test done while fully asleep. It will record data about your sleep patterns and may identify any sleep disorders. Electroencephalography (EEG) is carried out to measure the quality of sleep and detect seizures. Electromyography (EMG) is used to detect muscle movements including breathing muscles. Plethysmography bands are used to measure movements of the chest and abdomen. Electrocardiography (ECG) is done to detect abnormal heartbeats. Airflow probes are used to measure how much air moves through the airways. Pulse oximetry is carried out to measure heart rate and blood oxygen. A genetic blood test can be used to identify if the genetic mutation is responsible for Rett syndrome. If a change is found in the MECP2 gene, it can help confirm the diagnosis.

By Treatment:

Based on the treatment, the Rett syndrome therapeutics market can be bifurcated into

  • Physical Therapy
  • Occupational Therapy
  • Speech-Language Therapy
  • Behavioural Therapy
  • Nutritional Therapy
  • Physiotherapy
  • Antileptic Drugs
  • Beta-Blockers
  • Others

The Rett syndrome treatment focuses on managing the symptoms. Speech and language therapy, picture boards, eye gaze technology, and other visual aids to help with communication. If scoliosis does become established, a back brace and sometimes spinal surgery may be used to prevent the spine from curving further. An ankle-foot orthosis (lower leg brace) to help them walk independently. Occupational therapy to help develop the skills needed for dressing, feeding, and other daily activities. Medication for breathing and mobility problems, relieve indigestion and constipation. An anti-epileptic medicine to control seizures and beta-blocker drugs are given to control their heart rhythm.

By Distribution Channels:

In terms of distribution channel, the Rett syndrome therapeutics market can be classified into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

In terms of value, the hospital pharmacies accounted for the leading market share in 2020, which is attributable to the fact that the majority of the medications for Rett syndrome treatment are dispensed following by the prescription there and can so only be done by trained medical professionals. Hence, the hospital pharmacies segment is anticipated to enhance the market share over the forecast period.

Top Major Players

The UK Rett Syndrome therapeutics market is a lucrative market and the prominent players are focusing on organic growth strategies such as product launches, product approvals, and others such as patents and events. Additionally, the inorganic growth strategies activities witnessed in the market are acquisitions, and partnerships & collaborations. These activities have paved the way for the expansion of business and the customer base of market players. Major players operating in the market include Newron Pharmaceuticals Inc, GW Research Ltd, and Edison Pharmaceuticals Inc. among others.

Research Methodology

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

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or 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 develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

Our analysis methodology involves three critical stages:

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 around 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 the high level of accuracy and consistency. The market data is analysed and forecasted using appropriate statistical and coherent models. The report offers an overall analysis of the market size, growth, market share as well as 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:

  • Statistical Databases
  • Company Websites/Annual Reports
  • Trade Publications
  • Online Databases
  • Published Research Reports
  • Whitepapers
  • Press Releases of Key Market Players

Stage II: Market Data Analysis and Statistical Model

Market Trends: We generally look at macro parameter and micro indicators. The macro parameters includes changes in government policies, demand and supply of the market, government intervention programs and major market share. The micro indicators GDP growth, market size, market volume etc. We also understand nuances specific to each country like US, Canada, India, Germany, etc. and have worked across 60+ countries and hence no 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. start with large overall market and segment different areas and bottom-up approach i.e. start with population and epidemiology and roll up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form 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 North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. 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 the company level averages etc.

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

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed 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 are then, interpreted by considering following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
  • Analyzing current needs and determining penetration to estimate 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 its impact to 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 consumptions.
The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

To request a free sample copy this report, please complete the form below.

    We value your inquiry and offer free customization with every report to fulfil your exact research needs.

    This website is secure and your personal details are safe.

    This report addresses

    • Market intelligence to enable effective decision making
    • Growth opportunities and trend analysis
    • Competitive benchmarking
    • Historical data & forecasts
    • Regional opportunities

    Need more?

    • Speak to our analyst to know how this research was put together
    • Add more countries or regions as part of customisation
    • Understand how this report can have direct impact on your revenue

    Get a custom report based on your requirements