Duchenne Muscular Dystrophy Treatment Market: Introduction
- Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive degeneration of the muscles and weakness in them. It is a rare muscle disease, which largely affects men.
- Duchenne muscular dystrophy (DMD) therapeutics help treat this genetic disorder caused due to absence of dystrophin, a protein in the human body. Symptoms of the disease include weakness in the muscles of the hips, the thighs, the pelvic area, and the shoulders. Later, weakness is observed in the muscles of the arms, the legs, and the trunk. Various therapies and drugs to control DMD are available, which include mutation suppression, exon skipping, and steroid therapy.
- DMD is caused by changes (mutations) of the DMD gene on the X chromosome
Are you a Start-up willing to make it big in the Business? Grab an Exclusive PDF Brochure of Duchenne Muscular Dystrophy Treatment Market Report
Key Drivers and Restraints of Global Duchenne Muscular Dystrophy Treatment Market
- Increase in the number of patients with Duchenne muscular dystrophy is expected to propel the global Duchenne muscular dystrophy treatment market during the forecast period. According to the National Organization for Rare Disorders, one in 3,500 male babies born worldwide are affected with Duchenne muscular dystrophy. Overall, muscular dystrophies affect 250,000 individuals in the U.S.
- Increase in the number of clinical trials for testing the future treatment for Duchenne muscular dystrophy and wide product pipeline are likely to drive the global market for Duchenne muscular dystrophy treatment during the forecast period. Ataluren, casimersen, golodirsen, suvodirsen, and viltolarsen are five drug molecules undergoing Phase-3 clinical trial for their use in the treatment of Duchenne muscular dystrophy. EXONDYS 51 has been recently approved for treatment of the disease.
- Low rate of treatment caused by delayed diagnosis of the disease is restraining global Duchenne muscular dystrophy treatment market
Exon Skipping Segment to Witness Significant Growth
- Based on therapeutics, the global Duchenne muscular dystrophy treatment market can be segmented into mutation suppression, exon skipping, and steroid therapy
- The exon skipping segment accounted for the maximum share of the global Duchenne muscular dystrophy treatment market in 2018. The segment is expected to continue its dominance in the next few years, as a majority of exon skipping drugs are undergoing Phase-3 clinical trials. Only one drug approved for Duchenne muscular dystrophy i.e. Exondy 51 falls under the exon skipping therapeutics.
Hospital Pharmacies Segment to Witness Significant Growth
- In terms of distribution channel, the global Duchenne muscular dystrophy treatment market can be divided into hospital pharmacies, retail pharmacies, and online sales
- The hospital pharmacies segment generated the maximum revenues in 2018 and it is anticipated to witness significant growth during the forecast period. This can be attributed to growing number of patients with Duchenne muscular dystrophy being hospitalized. Hence, drugs for Duchenne muscular dystrophy are being sold on a large scale through hospital pharmacies.
North America to Dominate Global Duchenne Muscular Dystrophy Treatment Market
- In terms of region, the global Duchenne muscular dystrophy treatment market can be categorized into: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
- North America dominated the global Duchenne muscular dystrophy treatment market in 2018 and it is expected to maintain its leading position during the forecast period. The dominance of the region can be attributed to increasing research and development activities by major market players, expansion of product offerings by these players, and rise in the demand for early diagnosis in the region.
- The market in Asia Pacific is projected to register the maximum growth rate during the forecast period, owing to increase in the adoption of new products and rise in the incidence of Duchenne muscular dystrophy in the region
Expanding Operations in Future? To Get the Perfect Launch Ask for a Custom Duchenne Muscular Dystrophy Treatment Market Report
Key Manufacturers Operating in Global Market
The global Duchenne muscular dystrophy treatment market was highly fragmented in 2018. Key manufacturers operating in the global market are:
- Sarepta Therapeutics, Inc.
- Santhera Pharmaceuticals
- PTC Therapeutics
- NS Pharma, Inc.
- Wave Life Sciences
- Daiichi Sankyo Company
- Pfizer Inc.
- Solid Biosciences
Global Duchenne Muscular Dystrophy Treatment Market: Research Scope
Global Duchenne Muscular Dystrophy Treatment Market, by Therapeutics
- Mutation Suppression
- Exon Skipping
- Steroid Therapy
Global Duchenne Muscular Dystrophy Treatment Market, by Distribution Channel
- Hospital Pharmacies
- Retail Pharmacies
- Online Sales
Global Duchenne Muscular Dystrophy Treatment Market, by Region
- North America
- Rest of Europe
- Asia Pacific
- Australia & New Zealand
- Rest of Asia Pacific
- Latin America
- Rest of Latin America
- Middle East & Africa
- GCC Countries
- South Africa
- Rest of Middle East & Africa
This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.
Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:
- Customer Experience Maps
- Insights and Tools based on data-driven research
- Actionable Results to meet all the business priorities
- Strategic Frameworks to boost the growth journey
The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.
The following regional segments are covered comprehensively:
- North America
- Asia Pacific
- Latin America
- The Middle East and Africa
The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.
Below is a snapshot of these quadrants.
1. Customer Experience Map
The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.
2. Insights and Tools
The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.
3. Actionable Results
The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.
4. Strategic Frameworks
The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.
The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:
1. What can be the best investment choices for venturing into new product and service lines?
2. What value propositions should businesses aim at while making new research and development funding?
3. Which regulations will be most helpful for stakeholders to boost their supply chain network?
4. Which regions might see the demand maturing in certain segments in near future?
5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?
6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?
7. Which government regulations might challenge the status of key regional markets?
8. How will the emerging political and economic scenario affect opportunities in key growth areas?
9. What are some of the value-grab opportunities in various segments?
10. What will be the barrier to entry for new players in the market?
Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.