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Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2017

Published: Aug, 2017 | Pages: 119 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2017, provides an overview of the Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline landscape.

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. 

Report Highlights

Global Markets Direct's 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. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- 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 Buy

- 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 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 Genzyme Corp 25 Kowa Company Ltd 26 Neurotune AG 26 Novartis AG 27 Paratek Pharmaceuticals Inc 27 Recursion Pharmaceuticals Inc 28 Sarepta Therapeutics Inc 28 Scholar Rock Inc 29 Spotlight Innovation Inc 29 Translate Bio Inc 30 WAVE Life Sciences Ltd 30 Spinal Muscular Atrophy (SMA) - Drug Profiles 31 AAD-2004 - Drug Profile 31 ALB-111 - Drug Profile 32 Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 33 Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile 34 AVXS-101 - Drug Profile 35 branaplam - Drug Profile 41 CK-2127107 - Drug Profile 42 Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 48 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 49 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 50 K-828SP - Drug Profile 51 NT-1654 - Drug Profile 52 nusinersen - Drug Profile 53 olesoxime - Drug Profile 62 Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 67 Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Type 1 - Drug Profile 68 PMO-25 - Drug Profile 69 PTKSMA-1 - Drug Profile 70 RG-7916 - Drug Profile 71 Small Molecules 1 for Spinal Muscular Atrophy - Drug Profile 73 Small Molecules 2 for Spinal Muscular Atrophy - Drug Profile 74 Small Molecules for Central Nervous System Disorders - Drug Profile 75 Small Molecules for Spinal Muscular Atrophy - Drug Profile 76 Small Molecules for Spinal Muscular Atrophy - Drug Profile 77 Small Molecules for Spinal Muscular Atrophy - Drug Profile 78 Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 79 Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 80 Small Molecules to Inhibit GSK-3 for Spinal Muscular Atrophy - Drug Profile 81 SRK-015 - Drug Profile 82 STL-182 - Drug Profile 84 tirasemtiv - Drug Profile 85 Xcel-hNu - Drug Profile 96 Xcel-hNuP - Drug Profile 97 Spinal Muscular Atrophy (SMA) - Dormant Projects 98 Spinal Muscular Atrophy (SMA) - Discontinued Products 100 Spinal Muscular Atrophy (SMA) - Product Development Milestones 101 Featured News & Press Releases 101 Appendix 115 Methodology 115 Coverage 115 Secondary Research 115 Primary Research 115 Expert Panel Validation 115 Contact Us 115 Disclaimer 116
List of Tables
Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2017 Number of Products under Development by Companies, H2 2017 Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017 Number of Products under Development by Universities/Institutes, H2 2017 Products under Development by Companies, H2 2017 Products under Development by Companies, H2 2017 (Contd..1), H2 2017 Products under Development by Universities/Institutes, H2 2017 Number of Products by Stage and Target, H2 2017 Number of Products by Stage and Mechanism of Action, H2 2017 Number of Products by Stage and Route of Administration, H2 2017 Number of Products by Stage and Molecule Type, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by AveXis Inc, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Biogen Inc, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics Inc, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Genethon SA, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corp, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Kowa Company Ltd, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals Inc, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Recursion Pharmaceuticals Inc, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics Inc, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Scholar Rock Inc, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Spotlight Innovation Inc, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by Translate Bio Inc, H2 2017 Spinal Muscular Atrophy (SMA) - Pipeline by WAVE Life Sciences Ltd, H2 2017 Spinal Muscular Atrophy (SMA) - Dormant Projects, H2 2017 Spinal Muscular Atrophy (SMA) - Dormant Projects, H2 2017 (Contd..1), H2 2017 Spinal Muscular Atrophy (SMA) - Discontinued Products, H2 2017



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