Rett Syndrome - Pipeline Review, H2 2018

Rett Syndrome - Pipeline Review, H2 2018

Summary

The latest Pharmaceutical and Healthcare disease pipeline guide Rett Syndrome - Pipeline Review, H2 2018, provides an overview of the Rett Syndrome (Central Nervous System) pipeline landscape.

Rett syndrome is a neurodevelopmental disorder. This is caused by genetic mutation on MECP2 gene. Rett syndrome is described in four stages: Stage I or early onset, Stage II or rapid destruction, Stage III, or the plateau and Stage IV, or the late motor deterioration stage. Symptoms include slowed growth, loss of normal movement and coordination, loss of communication abilities, breathing problems, pain, irregular heartbeat and seizures. Risk factors include family history and gender. Treatment includes disease modifying drugs.

Report Highlights

The Pharmaceutical and Healthcare latest pipeline guide Rett Syndrome - Pipeline Review, H2 2018, provides comprehensive information on the therapeutics under development for Rett Syndrome (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 Rett Syndrome (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Rett Syndrome and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 4, 4, 9 and 2 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 5 molecules, respectively.

Rett Syndrome (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 Directs 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 Rett Syndrome (Central Nervous System).
- The pipeline guide reviews pipeline therapeutics for Rett Syndrome (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 Rett Syndrome (Central Nervous System) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Rett Syndrome (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 Rett Syndrome (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 Rett Syndrome (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 its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Rett Syndrome (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.

2
Introduction 4
Rett Syndrome - Overview 5
Rett Syndrome - Therapeutics Development 6
Rett Syndrome - Therapeutics Assessment 14
Rett Syndrome - Companies Involved in Therapeutics Development 22
Rett Syndrome - Drug Profiles 30
Rett Syndrome - Dormant Projects 109
Rett Syndrome - Product Development Milestones 110
Appendix 118

List Of Tables


Number of Products under Development for Rett Syndrome, H2 2018
Number of Products under Development by Companies, H2 2018
Number of Products under Development by Companies, H2 2018 (Contd..1), H2 2018
Number of Products under Development by Universities/Institutes, H2 2018
Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018 (Contd..1), H2 2018
Products under Development by Universities/Institutes, H2 2018
Number of Products by Stage and Target, H2 2018
Number of Products by Stage and Mechanism of Action, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Rett Syndrome - Pipeline by AMO Pharma Ltd, H2 2018
Rett Syndrome - Pipeline by Amorsa Therapeutics Inc, H2 2018
Rett Syndrome - Pipeline by Anavex Life Sciences Corp, H2 2018
Rett Syndrome - Pipeline by ArmaGen Inc, H2 2018
Rett Syndrome - Pipeline by AveXis Inc, H2 2018
Rett Syndrome - Pipeline by Biohaven Pharmaceutical Holding Company Ltd, H2 2018
Rett Syndrome - Pipeline by Eloxx Pharmaceuticals Inc, H2 2018
Rett Syndrome - Pipeline by GW Pharmaceuticals Plc, H2 2018
Rett Syndrome - Pipeline by Mitochon Pharmaceuticals Inc, H2 2018
Rett Syndrome - Pipeline by Mitsubishi Tanabe Pharma Corp, H2 2018
Rett Syndrome - Pipeline by Neuren Pharmaceuticals Ltd, H2 2018
Rett Syndrome - Pipeline by Neurolixis Inc, H2 2018
Rett Syndrome - Pipeline by Newron Pharmaceuticals SpA, H2 2018
Rett Syndrome - Pipeline by OPKO Health Inc, H2 2018
Rett Syndrome - Pipeline by Relmada Therapeutics Inc, H2 2018
Rett Syndrome - Pipeline by Ultragenyx Pharmaceutical Inc, H2 2018
Rett Syndrome - Dormant Projects, H2 2018

List Of Figures


Number of Products under Development for Rett Syndrome, H2 2018
Number of Products under Development by Companies, H2 2018
Number of Products by Top 10 Targets, H2 2018
Number of Products by Stage and Top 10 Targets, H2 2018
Number of Products by Top 10 Mechanism of Actions, H2 2018
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018

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