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Published: May, 2017 | Pages:
119 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Rett Syndrome - Pipeline Review, H1 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Rett Syndrome - Pipeline Review, H1 2017, 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 Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Rett Syndrome - Pipeline Review, H1 2017, 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, 7, 6 and 3 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 3 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 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 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 it's 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.
Table of Contents
List of Tables List of Figures Introduction Global Markets Direct Report Coverage Rett Syndrome - Overview Rett Syndrome - Therapeutics Development Pipeline Overview Pipeline by Companies Pipeline by Universities/Institutes Products under Development by Companies Products under Development by Universities/Institutes Rett Syndrome - Therapeutics Assessment Assessment by Target Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Rett Syndrome - Companies Involved in Therapeutics Development Amorsa Therapeutics Inc Anavex Life Sciences Corp Apteeus ArmaGen Inc Edison Pharmaceuticals Inc Eloxx Pharmaceuticals Ltd GW Pharmaceuticals Plc Horizon Pharma Plc Mitochon Pharmaceuticals Inc Mitsubishi Tanabe Pharma Corp Neuren Pharmaceuticals Ltd Neurolixis Inc Newron Pharmaceuticals SpA OPKO Health Inc Sage Therapeutics Inc Ultragenyx Pharmaceutical Inc Rett Syndrome - Drug Profiles amlexanox - Drug Profile Product Description Mechanism Of Action R&D Progress ANAVEX-273 - Drug Profile Product Description Mechanism Of Action R&D Progress BHV-5000 - Drug Profile Product Description Mechanism Of Action R&D Progress Bryostatin-1 - Drug Profile Product Description Mechanism Of Action R&D Progress CPT-157633 - Drug Profile Product Description Mechanism Of Action R&D Progress cysteamine DR - Drug Profile Product Description Mechanism Of Action R&D Progress desipramine hydrochloride - Drug Profile Product Description Mechanism Of Action R&D Progress ELX-02 - Drug Profile Product Description Mechanism Of Action R&D Progress fingolimod hydrochloride - Drug Profile Product Description Mechanism Of Action R&D Progress Gene therapy to Activate MECP2 for Rett Syndrome - Drug Profile Product Description Mechanism Of Action R&D Progress GWP-42006 - Drug Profile Product Description Mechanism Of Action R&D Progress LM-22A4 - Drug Profile Product Description Mechanism Of Action R&D Progress MP-101 - Drug Profile Product Description Mechanism Of Action R&D Progress NLX-101 - Drug Profile Product Description Mechanism Of Action R&D Progress Oligonucleotide for Rett Syndrome - Drug Profile Product Description Mechanism Of Action R&D Progress Recombinant Protein for Rett Syndrome - Drug Profile Product Description Mechanism Of Action R&D Progress SAGE-217 - Drug Profile Product Description Mechanism Of Action R&D Progress sarizotan hydrochloride - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecule to Agonize TrkB Receptor for Central Nervous System, Metabolic Disorders and ENT Disorders - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules for Pervasive Developmental Disorder and Rett Syndrome - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules to Antagonise NMDA Receptor for Rett Syndrome - Drug Profile Product Description Mechanism Of Action R&D Progress trofinetide - Drug Profile Product Description Mechanism Of Action R&D Progress UX-007 - Drug Profile Product Description Mechanism Of Action R&D Progress vatiquinone - Drug Profile Product Description Mechanism Of Action R&D Progress Rett Syndrome - Dormant Projects Rett Syndrome - Product Development Milestones Featured News & Press Releases Mar 22, 2017: Neuren's Phase 2 trial of trofinetide demonstrates significant clinical benefit in pediatric Rett syndrome Jan 30, 2017: Neuren completes Phase 2 trial in pediatric Rett syndrome Jan 19, 2017: Anavex Life Sciences Receives Grant From Rettsyndrome.org to Commence U.S. Phase 2 Trial in 2017 Nov 14, 2016: Neuren Completes Enrolment into Phase 2 Trial in Pediatric Rett Syndrome Aug 25, 2016: Neuren expands Phase 2 Rett syndrome pediatric clinical trial Jul 21, 2016: Newron Pharmaceuticals Announces Initiation of the STARS Study in the United States Jun 24, 2016: Neuren presents at the Rettsyndrome.org 2016 Research Symposium Jun 22, 2016: Neuren receives grant of new Australian patent for trofinetide to treat Autism Spectrum Disorders Jun 16, 2016: Newron Pharmaceuticals to Present STARS Trial Design at US Rett Syndrome Symposium Jun 06, 2016: Anavex Announces Positive Preclinical Results with ANAVEX 2-73 in Fragile X - Autism-Related Disorders May 20, 2016: Anavex Announces U.S. FDA Orphan Drug Designation to ANAVEX 2-73 for the Treatment of Rett Syndrome May 17, 2016: Mitochon Pharmaceuticals Raises $1.6M for Clinical Stage Development May 17, 2016: Newron Announces IND Approval for Sarizotan for Treatment of Rett Syndrome Apr 07, 2016: First subject commences Neuren's pediatric Rett syndrome Phase 2 trial Feb 25, 2016: Anavex Presents Preclinical Results of ANAVEX 2-73 in Rett Syndrome Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer
List of Tables
Number of Products under Development for Rett Syndrome, H1 2017 Number of Products under Development by Companies, H1 2017 Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017 Number of Products under Development by Universities/Institutes, H1 2017 Products under Development by Companies, H1 2017 Products under Development by Companies, H1 2017 (Contd..1), H1 2017 Products under Development by Universities/Institutes, H1 2017 Number of Products by Stage and Target, H1 2017 Number of Products by Stage and Mechanism of Action, H1 2017 Number of Products by Stage and Route of Administration, H1 2017 Number of Products by Stage and Molecule Type, H1 2017 Rett Syndrome - Pipeline by Amorsa Therapeutics Inc, H1 2017 Rett Syndrome - Pipeline by Anavex Life Sciences Corp, H1 2017 Rett Syndrome - Pipeline by Apteeus, H1 2017 Rett Syndrome - Pipeline by ArmaGen Inc, H1 2017 Rett Syndrome - Pipeline by Edison Pharmaceuticals Inc, H1 2017 Rett Syndrome - Pipeline by Eloxx Pharmaceuticals Ltd, H1 2017 Rett Syndrome - Pipeline by GW Pharmaceuticals Plc, H1 2017 Rett Syndrome - Pipeline by Horizon Pharma Plc, H1 2017 Rett Syndrome - Pipeline by Mitochon Pharmaceuticals Inc, H1 2017 Rett Syndrome - Pipeline by Mitsubishi Tanabe Pharma Corp, H1 2017 Rett Syndrome - Pipeline by Neuren Pharmaceuticals Ltd, H1 2017 Rett Syndrome - Pipeline by Neurolixis Inc, H1 2017 Rett Syndrome - Pipeline by Newron Pharmaceuticals SpA, H1 2017 Rett Syndrome - Pipeline by OPKO Health Inc, H1 2017 Rett Syndrome - Pipeline by Sage Therapeutics Inc, H1 2017 Rett Syndrome - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2017 Rett Syndrome - Dormant Projects, H1 2017
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