UK Duchenne Muscular Dystrophy (DMD) Drugs Market Report 2023 to 2030

UK Duchenne Muscular Dystrophy (DMD) Drugs Market Analysis

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UK Cystic Fibrosis Drugs Market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023 - 2030. The market for Duchenne Muscular Dystrophy (DMD) Drugs is expanding as a result of rising incidence and prevalence of duchenne muscular dystrophy, improved diagnosis of the disease, emergence of mutation specific and gene specific therapies, favourable government initiatives and supportive regulatory environments in various countries. Sarepta therapeutics, PTC therapeutics, Santhera pharmaceuticals are the top key market players operating in the Cystic fibrosis drugs market.

ID: IN10GBRD081 CATEGORY: Rare Diseases GEOGRAPHY: UK AUTHOR: Sakshi Mantri

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UK Duchenne Muscular Dystrophy (DMD) Drugs Market Analysis Summary

UK Duchenne Muscular Dystrophy (DMD) Drugs Market is valued at around $28.6 Mn in 2022 and is projected to reach $434.29 Mn by 2030, exhibiting a CAGR of 40.5% during the forecast period 2023-2030.

Duchenne muscular dystrophy is a severe disorder of a progressive kind of muscular dystrophy that mostly affects men, while there are very few instances in which it also affects women. The skeletal and cardiac muscles of people with Duchenne muscular dystrophy atrophy, or weaken, over time. Early signs of the illness include speech issues as well as a delayed capacity to stand, walk, or sit. The DMD therapeutics market has only three approved products - Exondys51, Translarna, and Emflaza. Increasing uptake of mutation-targeted therapies is likely to boost the sales of branded drugs. The exon-skipping platform is estimated to have an increased market share in future. Mutation-specific therapies, like Translarna, are projected to face limited adoption due to their high pricing. Associated adverse effects of steroid therapy make them a less favoured treatment option. The DMD market is projected to become intensely competitive in the medium and long term. Several novel mechanisms of action, such as myostatin inhibition, and gene therapy are under investigation. Other drug classes, such as exon-skipping and mutation-suppression are expected to gain over the forecast period, supported by rising adoption of these therapeutics.

Market Dynamics

Market Drivers

Rising incidence and prevalence of Duchenne muscular dystrophy, improved diagnosis of the disease, government awareness and patient advocacy programmes are market growth drivers.

Gene therapy advancements - Advancements in exon skipping and gene replacement therapies have generated interest in pharmaceutical companies and thus act as a market growth driver.

Collaborations and partnerships – Collaborations among research organizations, academic institutions and pharmaceutical companies act as a market growth driver.

Market Restraints

Stringent regulatory procedures and a lack of standardized protocol for the determination of clinical efficacy are market growth restraints

The high cost of Duchenne muscular dystrophy treatment is also a market growth restraint.

No cure for progressive disorder- DMD is a progressive disorder and its severity increases with time. Since DMD is a genetic disease there is no known cure and this acts as a market growth restraint.

Key players

Sarepta Therapeutics Pfizer BioMarin Pharmaceutical AveXis Solid Biosciences uniQure Ionis Pharmaceuticals Alnylam Pharmaceuticals Sangamo Therapeutics PTC Therapeutics

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 UK Duchenne Muscular Dystrophy (DMD) Drugs market

By Treatment:

Molecular therapies
Steroid therapies
Non- Steroidal Anti-inflammatory drugs (NSAID's)
Others

By Distribution Channel

Retail pharmacies and drug stores
Online pharmacies
Hospital pharmacies

By Route of Administration

Oral
Parenteral
Others

By End users

Hospitals
Home care settings
Clinics

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Market Overview
Market Growth Drivers & Restraints
Epidemiology of Disease Type
Market Segmentation
Market Share
Competitive Landscape
Key Company Profiles
Healthcare Policies & Regulatory Framework
Reimbursement Scenario
Factors Driving Future Growth

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Establishing market drivers and trends
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