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

US Duchenne Muscular Dystrophy (DMD) Drugs Market Analysis

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US 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: IN10USRD081 CATEGORY: Rare Diseases GEOGRAPHY: US AUTHOR: Sakshi Mantri

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US Duchenne Muscular Dystrophy (DMD) Drugs Market Analysis Summary

US Duchenne Muscular Dystrophy (DMD) Drugs Market is valued at around $0.47 Bn in 2022 and is projected to reach $7.02 Bn 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, Inc. Pfizer Inc. PTC Therapeutics, Inc. Santhera Pharmaceuticals Catabasis Pharmaceuticals, Inc. NS Pharma, Inc. Solid Biosciences Inc. Wave Life Sciences Ltd. FibroGen, Inc. Capric

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 US 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

Methodology for Database Creation

Our database offers a comprehensive list of healthcare centers, meticulously curated to provide detailed information on a wide range of specialties and services. It includes top-tier hospitals, clinics, and diagnostic facilities across 30 countries and 24 specialties, ensuring users can find the healthcare services they need.

Additionally, we provide a comprehensive list of Key Opinion Leaders (KOLs) based on your requirements. Our curated list captures various crucial aspects of the KOLs, offering more than just general information. Whether you're looking to boost brand awareness, drive engagement, or launch a new product, our extensive list of KOLs ensures you have the right experts by your side. Covering 30 countries and 36 specialties, our database guarantees access to the best KOLs in the healthcare industry, supporting strategic decisions and enhancing your initiatives.

How Do We Get It?

Our database is created and maintained through a combination of secondary and primary research methodologies.

1. Secondary Research

With many years of experience in the healthcare field, we have our own rich proprietary data from various past projects. This historical data serves as the foundation for our database. Our continuous process of gathering data involves:

Analyzing historical proprietary data collected from multiple projects.
Regularly updating our existing data sets with new findings and trends.
Ensuring data consistency and accuracy through rigorous validation processes.

With extensive experience in the field, we have developed a proprietary GenAI-based technology that is uniquely tailored to our organization. This advanced technology enables us to scan a wide array of relevant information sources across the internet. Our data-gathering process includes:

Searching through academic conferences, published research, citations, and social media platforms
Collecting and compiling diverse data to build a comprehensive and detailed database
Continuously updating our database with new information to ensure its relevance and accuracy

2. Primary Research

To complement and validate our secondary data, we engage in primary research through local tie-ups and partnerships. This process involves:

Collaborating with local healthcare providers, hospitals, and clinics to gather real-time data.
Conducting surveys, interviews, and field studies to collect fresh data directly from the source.
Continuously refreshing our database to ensure that the information remains current and reliable.
Validating secondary data through cross-referencing with primary data to ensure accuracy and relevance.

Combining Secondary and Primary Research

By integrating both secondary and primary research methodologies, we ensure that our database is comprehensive, accurate, and up-to-date. The combined process involves:

Merging historical data from secondary research with real-time data from primary research.
Conducting thorough data validation and cleansing to remove inconsistencies and errors.
Organizing data into a structured format that is easily accessible and usable for various applications.
Continuously monitoring and updating the database to reflect the latest developments and trends in the healthcare field.

Through this meticulous process, we create a final database tailored to each region and domain within the healthcare industry. This approach ensures that our clients receive reliable and relevant data, empowering them to make informed decisions and drive innovation in their respective fields.