Genetic Disorders Drugs Market report which is an end to end market guide. This report includes historic, current and future market scenario giving the reader a holistic as well as an atomistic view of the industry. The Genetic Disorders Drugs market research report anticipates the industry growth curve to go uphill under the study period of 2013–2023. The consumption, sales, revenue, gross margins are collected and analyzed at the regional level across the value chain.
Global Genetic Disorders Drugs Market to 2023 A Rapidly Growing Treatment Landscape Driven by Targeted Complement System Inhibitors and Enzyme Replacement Therapies to Treat PNH and Lysosomal Storage Disease
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Summary
The genetic disorders therapy area consists of indications that arise because of an abnormality in a person’s DNA. This report focuses on the key therapy area indications of cystic fibrosis CF, Duchenne muscular dystrophy DMD, lysosomal storage disease LSD and paroxysmal nocturnal hemoglobinuria PNH.
Genetic disorders are frequently inherited from an individual’s parents but can also arise because of a new de novo mutation. The treatment landscape for genetic disorders is dominated commercially by orphan drugs for the treatment of rare genetic disorders.
Overall, 1,390 products are being actively developed in the genetic disorders pipeline. The pipelines of CF, DMD, LSD and PNH contain 163, 109, 174 and 24 products, respectively. The pipeline is dominated by targeted therapies that aim to treat the underlying cause, which is often specific to the particular indication.
Scope
Global revenue from the genetic disorders market is forecast to increase from $19.6 billion in 2016 to $47.7 billion in 2023, at a compound annual growth rate of 13.55%. What is driving this growth?
The leading companies in terms of market share are Alexion, Sanofi, Shire and Vertex. Which of these are forecast to experience the largest growth?
There are new approvals and latestage products set to enter the market during the forecast period. Which drugs will achieve blockbuster status?
The market has been dominated by complement system inhibitors and enzyme replacement therapies. To what extent will these classes of drugs and others dominate the market over the forecast period?
There are 1,390 genetic disorders products in the pipeline. What molecular targets are most abundant in the pipeline and what role will pipeline product approvals play in market growth?
Genetic disorders clinical trials have an overall attrition rate of around 93%, what can companies do to maximize their chance of success?
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Key questions answered in this report
• What will the market size be in 2023 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?
Table of Contents
1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 7
1.2 List of Figures 8
2 Introduction 11
2.1.1 Cystic Fibrosis 12
2.1.2 Duchenne Muscular Dystrophy 12
2.1.3 Lysosomal Storage Disease 12
2.1.4 Paroxysmal Nocturnal Hemoglobinuria 14
2.2 Symptoms 14
2.3 Diagnosis 15
2.3.1 Cystic Fibrosis 17
2.3.2 Duchenne Muscular Dystrophy 18
2.3.3 Lysosomal Storage Disease 19
2.3.4 Paroxysmal Nocturnal Hemoglobinuria 19
2.4 Etiology and Pathophysiology 20
2.4.1 Cystic Fibrosis 20
2.4.2 Duchenne Muscular Dystrophy 21
2.4.3 Lysosomal Storage Disease 22
2.4.4 Paroxysmal Nocturnal Hemoglobinuria 25
2.5 Epidemiology Patterns and Forecasts Prevalence and Patient Segmentation 26
2.5.1 Cystic Fibrosis 27
2.5.2 Duchenne Muscular Dystrophy 28
2.5.3 Lysosomal Storage Disease 28
2.5.4 Paroxysmal Nocturnal Hemoglobinuria 29
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