UK Duchenne Muscular Dystrophy (DMD) Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2589

  • Format: PPT, PDF

UK Duchenne Muscular Dystrophy Therapeutics Market: Segmented by Drugs, Therapeutics, and Distribution Channels – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

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SKU: 2589 Categories: ,

Report Overview

The UK Duchenne Muscular Dystrophy 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.

Duchenne Muscular Dystrophy, or DMD, is a debilitating genetic condition that causes a gradual loss of muscle function that affects everyday movements and activities. Muscular dystrophy is a condition that causes progressive wasting of the muscles. Duchenne muscular dystrophy is a particular type of muscular dystrophy caused by a mutation in the DMD gene. It affects more boys than girls. The DMD gene helps produce a protein called dystrophin, which is important for muscle strength, support, and repair. People with Duchenne muscular dystrophy don’t produce the normal form of dystrophin, which means their muscles are more easily damaged and don’t work properly. The genetic mutation of the DMD gene is either inherited from parents or caused by a genetic change in the child. This is the most common type affecting children.

DMD is caused by a genetic fault on the X chromosome and mainly affects boys because males only have one X chromosome. Females have two X chromosomes, so may carry the defective gene without showing any symptoms. It is usually recognized between three and six years of age. DMD is an X-linked recessive disorder caused by genetic variants in the dystrophin gene on the X chromosome. The incidence rate of DMD is 10.7 to 27.8 per 100,000 newborn males. In the UK, about 100 boys are born with DMD each year, and there are about 2,500 people living with the condition at any one time.

Market Growth Drivers

Major driving factors in the Duchenne Muscular Dystrophy market are the introduction of novel drugs and therapies, disease-modifying therapies, strong support from various companies for drug development. Moreover, mutation-specific therapies are likely to emerge as a major breakthrough in the Duchenne muscular dystrophy treatment.

Drug developers are also focusing on developing new drugs and therapies targeting newborns, young infants, and also last-stage DMD patients. In the last few years, there has been rapid development in Duchenne muscular dystrophy therapeutics.

According to studies, the general deterioration in mental health in broad terms due to a variety of COVID-19 factors with feelings of anxiousness, disappointment, and rising frustration which is certainly a factor that will propel the DMD treatment market up.

Market Restraints

However, there is still a large number of patients who are not able to benefit from therapies and drugs due to its high cost and lack of awareness may hamper the DMD treatment market over the forecast period.

TABLE OF CONTENTS

1. UK Duchenne Muscular Dystrophy Therapeutics Market Overview…………….
A. Market Size
2. Market Growth Drivers & Restraints………………………………………………….
Market Growth Drivers
A. Introduction of Novel Drugs and Technological Advancements
B. Mutation-specific Therapies Emerges as a Major Breakthrough
C. Strong Support from Various Companies for Drug Development
Market Restraints
D. High Therapy Costs & Low Treatment Rates
E. Lack of Awareness among Individuals
3. Major Types of Duchenne Muscular Dystrophy…………………….
A. Congenital Duchenne Muscular Dystrophy
B. Genetic Duchenne Muscular Dystrophy
4. UK Duchenne Muscular Dystrophy Therapeutics Market Segmentation………….
A. By Drugs
I. Translarna
II. Corticosteroids
III. NSAIDs
IV. Other Drugs
B. By Therapeutics
I. Mutation Suppression Therapy
II. Exon Skipping approach
III. Stem cell therapy
IV. Other therapies
C. By Distribution Channel
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. UK Duchenne Muscular Dystrophy Major Products 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. PTC Company overview, Product & Services, Strategies & Financials
B. Roche Company overview, Product & Services, Strategies & Financials
C. Sarepta 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 DMD Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Drugs:

Based on the drugs, the DMD treatment market can be segmented into

  • Translarna
  • Corticosteroids
  • NSAIDs
  • Other drugs

Ataluren, sold under the brand name Translarna, medication for the treatment of DMD, designed by PTC Therapeutics. Moreover, corticosteroids are likely to emerge as the top-selling products. Various scientific research has found that compared to various products to treat Duchenne Muscular Dystrophy, corticosteroids help in improving muscle strength for up to two to five years, thus further anticipated to increase the market share over the forecast period.

By Therapeutics:

Based on the therapeutics, the DMD treatment market can be bifurcated into

  • Mutation Suppression therapy
  • Exon Skipping approach
  • Stem cell therapy
  • Other therapies

Mutation suppression therapy in Duchenne Muscular Dystrophy targets the specific mutation causing the muscular dystrophy. Therefore, Mutation suppression therapy is likely to be one of the highly preferred therapy for Duchenne Muscular Dystrophy treatment over the forecast period.

By Distribution Channel:

In terms of distribution channel, the DMD treatment market can be classified into

  • Hospital pharmacies
  • Rectal pharmacies
  • Online pharmacies

An increasing number of people are being hospitalized for Duchenne Muscular Dystrophy. Hence, drugs for Duchenne Muscular Dystrophy are being sold on a large scale through hospital pharmacies. Therefore, hospital pharmacies are anticipated to be the largest distribution channel between the 2020 and 2028 forecast periods.

Top Major Players

The reports cover all the key developments in the DMD treatment market as organic and inorganic growth strategies. Various companies are focusing on organic growth strategies such as product launches, product approvals, and others such as patents and events. Inorganic growth strategies activities witnessed in the market were acquisitions and partnership & collaborations. These activities have paved the way for the expansion of the business and the customer base of market players. Major prominent players operating in the DMD market include PTC Therapeutics Ltd, Hoffmann-La Roche Ltd, GlaxoSmithKline UK Ltd, Sarepta Therapeutics Inc., and Prosensa Therapeutics B.V. 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

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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.

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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)

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  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
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The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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