Global Spinal Muscular Atrophy (SMA) Industry Trends, Regulations And Competitive Landscape Outlook H2 2017

Overview

Spinal muscular atrophy (SMA) is a genetic disease that attacks nerve cells, called motor neurons, in the spinal cord. These critically important cells are responsible for supplying electrical and chemical messages to muscle cells. Without the proper input from the motor neurons, muscle cells cannot function properly. The muscle cells will, therefore, become much smaller (atrophy) and will produce symptoms of muscle weakness. This can affect walking, crawling, breathing, swallowing, and head and neck control.

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Report Highlights

The Pharmaceutical and Healthcare latest pipeline guide Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (SMA) (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Spinal Muscular Atrophy (SMA) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 2, 4, 1, 13 and 4 respectively. Major Scope of the report:

- The pipeline guide provides a snapshot of the global therapeutic landscape of Spinal Muscular Atrophy (SMA) (Central Nervous System). 
- The pipeline guide reviews pipeline therapeutics for Spinal Muscular Atrophy (SMA) (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources. 
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. 
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. 

Table of Contents 

Table of Contents 2 
List of Tables 4 
List of Figures 4 
Introduction 5 
Global Markets Direct Report Coverage 5 
Spinal Muscular Atrophy (SMA) - Overview 6 
Spinal Muscular Atrophy (SMA) - Therapeutics Development 7 
Pipeline Overview 7 
Pipeline by Companies 8 
Pipeline by Universities/Institutes 11 
Products under Development by Companies 12 
Products under Development by Universities/Institutes 14 
Spinal Muscular Atrophy (SMA) - Therapeutics Assessment 15 
Assessment by Target 15 
Assessment by Mechanism of Action 17 
Assessment by Route of Administration 19 
Assessment by Molecule Type 21 
Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development 23 
AveXis Inc 23 
Biogen Inc 23 
Cytokinetics Inc 24 
F. Hoffmann-La Roche Ltd 24 
Genethon SA 25  

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