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Explore spinal muscular atrophy (SMA) pharmaceutical and healthcare pipeline review H2

Spinal muscular atrophy (SMA) Pharmaceutical and Healthcare Pipeline Review H2

Pune, India, 17th August 2017: WiseGuyReports announced addition of new report, titled “Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2017”.


Spinal muscular atrophy Make an Inquiry about this news (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

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.

Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 6 and 2 molecules, respectively.

Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.


- 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.

- The pipeline guide reviews key companies involved in Spinal Muscular Atrophy (SMA) (Central Nervous System) therapeutics and enlists all their major and minor projects.

- The pipeline guide evaluates Spinal Muscular Atrophy (SMA) (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

- The pipeline guide reviews latest news related to pipeline therapeutics for Spinal Muscular Atrophy (SMA) (Central Nervous System)

Reasons to access

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

- Find and recognize significant and varied types of therapeutics under development for Spinal Muscular Atrophy (SMA) (Central Nervous System).

- Classify potential new clients or partners in the target demographic.

- Develop tactical initiatives by understanding the focus areas of leading companies.

- Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.

- Formulate corrective measures for pipeline projects by understanding Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline depth and focus of Indication therapeutics.

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.

- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Content: Key Points

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

  1. Hoffmann-La Roche Ltd 24

Genethon SA 25

Genzyme Corp 25

Kowa Company Ltd 26

Neurotune AG 26



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