Spinal Muscular Atrophy (SMA) Therapeutics Pipeline Analysis 2017 - Clinical Trials & Results, Patent, Designation, Collaboration, and Other Developments

  • ID: 4340275
  • Report
  • 86 pages
  • P&S Market Research
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Disease Overview

Spinal muscular atrophy is a rare inherited genetic disease that is characterized by loss of nerve cells, known as motor neurons. Motor neurons receive the nerve impulses, which are transmitted from the brain to the spinal cord and, further transmits these impulses to the muscles with the help of peripheral nerves. The loss of motor neurons can lead to atrophy and weakness in the muscles, that are closest to the trunk of the body such as back, hips and shoulders. Spinal muscular atrophy is mainly caused due to the mutation in the SMA2 and SMN1 genes in chromosome. Any mutation in SMA1 gene can lead to spinal muscular atrophy.

According to National Organization for Rare Disorders, around 95% to 98% people who suffer from spinal muscular atrophy have deletions in the SMA1 (Survival of Motor Neuron 1) gene that results in a decreased production of SMN protein. The prevalence of spinal muscular atrophy has increased in the past few years, due to various risk factors such as smoking, family history, lack of awareness and delay in diagnosis. According to National Organization for Rare Disorders, the incidence of spinal muscular atrophy is approximately one in 10,000 live births. The disease is categorized into four types namely, Type I, Type II, Type III and Type IV. SMA Type I occurs mainly in infants aged between three to six months, with symptoms including very little muscle tone, weak muscles and feeding and breathing problems. SMA Type II and III both are very severe type of spinal muscular atrophy, and affects infants aged between six months to two years. SMA Type IV mainly affects the adult population. Some of the symptoms associated with spinal muscular atrophy include weakness in muscles, breathing difficulty, lack of head control, feeding difficulty and floppy infant (poor muscle tone).

Pipeline Analysis

As of June 2017, the spinal muscular atrophy pipeline comprises approximately 16 active drug candidates in different stages of development.

Competitive Landscape

Some of the other key players developing drugs for the treatment of spinal muscular atrophy include Cytokinetics, Inc., F. Hoffman La-Roche Ltd, WAVE Life Sciences Ltd. and others.

Scope for Customization

Customization is offered as per specific business requirements of clients. Illustrative customization within the scope of this report includes:
  • Market Forecast - Market analysis and forecast for the drug candidates in the latest stage of development
  • Company Profiles - Wider company coverage in terms of detailed analysis or additional company profiles
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1. Research Background
1.1 Research Objectives
1.2 Definition
1.3 Research Scope
1.4 Key Stakeholders

2. Research Methodology
2.1 Secondary Research
2.2 Primary Research

3. Executive Summary

4. Pipeline Outlook
4.1 Overview
4.2 Key Drivers
4.3 Key Barriers
4.4 Spinal Muscular Atrophy Pipeline Analysis
4.4.1 Pipeline Analysis By Phase
4.4.2 Pipeline Analysis By Molecule Type
4.4.3 Pipeline Analysis By Route Of Administration
4.4.4 Pipeline Analysis By Company

5. Spinal Muscular Atrophy Therapeutics Pipeline Analysis By Phase (2017)
5.1 Phase Iii: Drug Profiles
5.1.1 Pre-Clinical Study
5.1.2 Pre-Clinical Results
5.1.3 Clinical Trials
5.1.4 Clinical Results
5.1.5 Strategic Development
5.1.6 Designation
5.1.7 Grants
5.1.8 Patent
5.1.9 Technology
5.2 Phase Ii: Drug Profiles
5.2.1 Pre-Clinical Study
5.2.2 Pre-Clinical Results
5.2.3 Clinical Trials
5.2.4 Clinical Results
5.2.5 Strategic Development
5.2.6 Designation
5.2.7 Grants
5.2.8 Patent
5.2.9 Technology
5.3 Phase I: Drug Profiles
5.3.1 Pre-Clinical Study
5.3.2 Pre-Clinical Results
5.3.3 Clinical Trials
5.3.4 Clinical Results
5.3.5 Strategic Development
5.3.6 Designation
5.3.7 Grants
5.3.8 Patent
5.3.9 Technology
5.4 Pre-Clinical: Drug Profiles
5.4.1 Pre-Clinical Study
5.4.2 Pre-Clinical Results
5.4.3 Strategic Development
5.4.4 Designation
5.4.5 Grants
5.4.6 Patent
5.4.7 Technology
5.5 Discovery: Drug Profiles
5.5.1 Strategic Development
5.5.2 Designation
5.5.3 Grants
5.5.4 Patent
5.5.5 Technology

6 Competitive Landscape
6.1 Key Players Benchmarking For Spinal Muscular Atrophy Drug Candidates
6.2 Swot Analysis Of Spinal Muscular Atrophy Pipeline

7 Company Profiles
7.1 Business Overview
7.2 Product And Service Offerings

8 Appendix
8.1 Abbreviations

List Of Tables
Pipeline Analysis Of Spinal Muscular Atrophy, By Company (2017)
Description Of Phase Iii Drug Candidates
Clinical Trials Of Phase Iii Drug Candidates
Description Of Phase Ii Drug Candidates
Clinical Trials Of Phase Ii Drug Candidates
Description Of Phase I Drug Candidates
Clinical Trials Of Phase I Drug Candidates
Pre-Clinical Drug Candidates
Discovery Drug Candidates
Companies- At A Glance

List Of Figures
Research Methodology For The Spinal Muscular Atrophy Pipeline Analysis
Split Of Primary And Secondary Research
Breakdown Of Primary Research Respondents, By Industry Participant
Breakdown Of Primary Research Respondents, By Company Type
Number Of Spinal Muscular Atrophy Drug Candidates Under Development (2017)
Spinal Muscular Atrophy Pipeline Split, By Molecule Type (2017)
Spinal Muscular Atrophy Pipeline Split, By Route Of Administration (2017)
Key Players Benchmarking For Spinal Muscular Atrophy Drug Candidates
Swot Analysis Of Spinal Muscular Atrophy Pipeline
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