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

Research and Markets
Posted on: 03 Aug 17

The "Spinal Muscular Atrophy (SMA) Therapeutics Pipeline Analysis 2017 - Clinical Trials & Results, Patent, Designation, Collaboration, and Other Developments" report has been added to Research and Markets' offering.

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.

Key players are (development drugs for the treatment of spinal muscular atrophy)

  • Cytokinetics, Inc.,
  • F. Hoffman La-Roche Ltd,
  • WAVE Life Sciences Ltd.
  • and others.

Key Topics Covered:

1. Research Background

2. Research Methodology

3. Executive Summary

4. Pipeline Outlook

5. Spinal Muscular Atrophy Therapeutics Pipeline Analysis By Phase (2017)

6 Competitive Landscape

7 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/research/w3xc34/spinal_muscular

View source version on businesswire.com: http://www.businesswire.com/news/home/20170803005597/en/

Business Wire
www.businesswire.com

Last updated on: 03/08/2017

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