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Global Duchenne Muscular Dystrophy Drugs Market 2015-2019

  • August 2015
  • -
  • Infiniti Research Limited
  • -
  • 67 pages

About Duchenne muscular dystrophy
Duchenne muscular dystrophy is an X-linked disease, which generally affects the male population. It is characterized by progressive muscle degeneration and weakness. The disease is caused due to mutations in the dystrophin gene, which result in the lack of production of dystrophin (a protein essential for maintaining healthy muscle function) in individuals. The lack of dystrophin weakens muscle function, causes loss of ambulation, harms the respiratory and cardiac function, and ultimately leads to death. The main symptoms of this condition appears in childhood and includes delay of motor functions; progressive muscle weakness; muscle contractures in legs; and muscle weakness in arms, neck, and more severely in the lower half of the body. The bones also develop peculiarly, causing skeletal deformities in the patient. Occurrences of pseudo-hypertrophy, cardiomyopathy, and breathing complications are some of the other symptoms of this condition.

Technavio's analysts forecast the global Duchenne muscular dystrophy drugs market to grow at a CAGR of 152.3% over the period 2014-2019.

Covered in this report
This report covers the present scenario and the growth prospects of the global Duchenne muscular dystrophy drugs market for the period 2015-2019. To calculate the market size, the report considers revenue generated from the sales of various off-label and branded drugs and the expected launch of drug candidates intended to be used in the treatment of Duchenne muscular dystrophy.

Based on mechanism of action of the drugs, the market is segmented as follows:
- Exon skipping mechanism
- Premature stop codon red through mechanism
- Others
This report includes a discussion of the market in the following three regions:
- Americas: the US, Canada, Mexico, and Brazil
- EMEA: the UK, Germany, Italy, France, Spain and Middle Eastern and African countries such as Israel, South Africa, Egypt, Sudan, Kuwait, Saudi Arabia, Qatar, and the UAE
- APAC: Japan, China, Australia, Singapore, South Korea, and India.

The report also presents the vendor landscape and a corresponding detailed analysis of the top vendors in the market. The vendor landscape section includes a market share analysis of major vendors along with the competitive performances of their product portfolios. In addition, the report discusses the major drivers that influence the growth of the market. It also outlines the challenges faced by the vendors and the market at large, as well as the key trends emerging in the market.

Technavio's report, Global Duchenne Muscular Dystrophy Drugs Market 2015-2019, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the Americas, APAC, and EMEA; it also covers the landscape of the global Duchenne muscular dystrophy drugs market and its growth prospects in the coming years. The report includes a discussion of the key vendors operating in this market.
Key regions
- Americas
- APAC
- EMEA
Key Vendors
- BioMarin Pharmaceutical
- Eli Lilly
- PTC Therapeutics
- Santhera Pharmaceuticals
- Sarepta Therapeutics
Other prominent vendors

- Acceleron Pharma
- Akashi Therapeutics
- Asklepios BioPharmaceuticals
- Bristol-Myers Squibb
- Capricor Therapeutics
- Catabasis Pharmaceuticals
- Janssen Pharmaceuticals
- Lexicon Pharmaceuticals
- Marathon Pharmaceuticals
- Nippon Shinyaku
- Nobelpharma
- Pfizer
- Summit Therapeutics
- Taiho Pharmaceutical

Market driver
- Significant unmet medical need
- For a full, detailed list, view our report
Market challenge
- High cost
- For a full, detailed list, view our report
Market trend
- Focus on novel targets
- For a full, detailed list, view our report

Key Questions Answered in this Report
- What will the market size be in 2019 and what will the growth rate be?
- What are the key market trends?
- What is driving this market?
- What are the challenges to market growth?
- Who are the key vendors in this market space?
- What are the market opportunities and threats faced by the key vendors?
- What are the strengths and weaknesses of the key vendors?

Table Of Contents

Global Duchenne Muscular Dystrophy Drugs Market 2015-2019
PART 01: Executive summary
Highlights
PART 02: Scope of the report
Market overview
Top-vendor offerings
PART 03: Market research methodology
Research methodology
Economic indicators
PART 04: Introduction
Key market highlights
PART 05: Market landscape
Market overview
Market size and forecast
Five forces analysis
PART 06: Market segmentation by mechanism of action
Exon skipping mechanism
Premature stop codon read-through mechanism
Others
PART 07: Understanding the disorder
Etiology
Symptoms
Biomarkers
Diagnosis
Staging
Traditional methods of management of Duchenne muscular dystrophy
Recent developments in Duchenne muscular dystrophy therapy
PART 08: Duchenne muscular dystrophy: epidemiology and cost analysis
Epidemiology
Burden associated with Duchenne muscular dystrophy
PART 09: Geographical segmentation
PART 10: Pipeline portfolio
PART 11: Market drivers
PART 12: Impact of drivers
PART 13: Market challenges
PART 14: Impact of drivers and challenges
PART 15: Market trends
PART 16: Vendor landscape
Competitive scenario
Market share analysis 2014
Other and future prominent vendors
PART 17: Key vendor analysis
BioMarin Pharmaceutical
Lilly
PTC Therapeutics
Santhera Pharmaceuticals
Sarepta Therapeutics
PART 18: Appendix
List of abbreviation
PART 19: Explore Technavio



List of Exhibits
Exhibit 01: Global Duchenne muscular dystrophy drugs market 2014-2019 ($ millions)
Exhibit 02: Five forces analysis
Exhibit 03: Heredogram of Duchenne muscular dystrophy
Exhibit 04: Biomarkers used in Duchenne muscular dystrophy
Exhibit 05: Various approaches to treating Duchenne muscular dystrophy
Exhibit 06: Percentage of Duchenne muscular dystrophy patients treated by exon skipping therapy
Exhibit 07: Prevalence of nonsense mutation Duchenne muscular dystrophy 2014
Exhibit 08: Global Duchenne muscular dystrophy drugs market segmentation by geography 2014
Exhibit 09: Duchenne muscular dystrophy: Pipeline portfolio
Exhibit 10: Impact of drivers
Exhibit 11: Impact of drivers and challenges
Exhibit 12: BioMarin Pharmaceutical: Key takeaways
Exhibit 13: Lilly: Key takeaways
Exhibit 14: PTC Therapeutics: Key takeaways
Exhibit 15: Santhera Pharmaceuticals: Key takeaways
Exhibit 16: Sarepta Therapeutics: Key takeaways

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