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肌強直性營養不良:開發平台分析

Myotonic Dystrophy - Pipeline Review, H2 2016

出版商 Global Markets Direct 商品編碼 271823
出版日期 內容資訊 英文 60 Pages
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肌強直性營養不良:開發平台分析 Myotonic Dystrophy - Pipeline Review, H2 2016
出版日期: 2016年07月20日 內容資訊: 英文 60 Pages
簡介

肌強直性營養不良為遺傳性的肌肉萎縮症之一,帶給肌肉和人體整體不良影響。主要的症狀為呼吸困難和吞嚥障礙、消化障礙、白天過度嗜睡、學習障礙等。主要的治療方法為外科手術和皮質類固醇劑等。

本報告提供全球各國的肌強直性營養不良治療用的開發中產品的開發情形相關分析、產品開發與上市的最新趨勢,及各臨床實驗階段的產品一覽,主要企業簡介,主要藥物概要,最新的產業趨勢等資訊,為您概述為以下內容。

目錄

簡介

  • 分析範圍

肌強直性營養不良概要

治療藥的開發

  • 肌強直性營養不良的開發中產品:概要
  • 肌強直性營養不良的開發中產品:比較分析

各企業開發中的肌強直性營養不良治療藥

大學/研究機關研究中的肌強直性營養不良治療藥

開發中產品的概要

  • 臨床實驗階段的產品
  • 初期階段的產品

肌強直性營養不良治療藥:開發中的產品一覽(各企業)

肌強直性營養不良治療藥:研究中的產品一覽(各大學/研究機關)

開發肌強直性營養不良治療藥的企業

  • BioMarin Pharmaceutical Inc.
  • F. Hoffmann-La Roche Ltd.
  • Genzyme Corporation
  • Isis Pharmaceuticals, Inc.
  • Marina Biotech, Inc.
  • Valentia Biopharma S.L.

肌強直性營養不良:治療藥的評估

  • 單劑治療藥的情況
  • 各標靶
  • 各作用機制
  • 各投藥法
  • 各分子類型

藥物簡介

  • 脊髓性肌萎縮症、肌強直性營養不良的反義寡核苷酸
    • 產品概要
    • 功能機制
    • 研究開發(R&D)的發展情形
  • ISIS-DMPKRx
  • PRO-135
  • 肌強直性營養不良的RNA干擾寡核苷酸
  • 以CUG RNA為標的之肌強直性營養不良第一型醫藥品
  • 以RNA為標的之肌強直性營養不良小分子
  • 肌強直性營養不良的第一型醫藥品
  • 以RNA為標的之肌強直性營養不良小分子
  • SRT-149
  • SRT-152
  • VAL-0411
  • VLT-001
  • VLT-002
  • VLT-003
  • VLT-004
  • VLT-005
  • VLT-014
  • VLT-015
  • VLT-016
  • VLT-017
  • VLT-025

肌強直性營養不良治療藥:開發中產品的最新趨勢

肌強直性營養不良相關產品的開發里程碑

  • 值得注意的最新趨勢、新聞稿

附錄

圖表一覽

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目錄
Product Code: GMDHC8295IDB

Summary

Global Markets Direct's, 'Myotonic Dystrophy - Pipeline Review, H2 2016', provides an overview of the Myotonic Dystrophy pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Myotonic Dystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Myotonic Dystrophy and features dormant and discontinued projects.

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

  • The report provides a snapshot of the global therapeutic landscape of Myotonic Dystrophy
  • The report reviews pipeline therapeutics for Myotonic Dystrophy by companies and universities/research institutes based on information derived from company and industry-specific sources
  • The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved Myotonic Dystrophy therapeutics and enlists all their major and minor projects
  • The report assesses Myotonic Dystrophy therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type
  • The report summarizes all the dormant and discontinued pipeline projects
  • The report reviews latest news related to pipeline therapeutics for Myotonic Dystrophy

Reasons to buy

  • Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Identify and understand important and diverse types of therapeutics under development for Myotonic Dystrophy
  • Identify potential new clients or partners in the target demographic
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
  • Devise corrective measures for pipeline projects by understanding Myotonic Dystrophy 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
  • Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Myotonic Dystrophy Overview
  • Therapeutics Development
    • Pipeline Products for Myotonic Dystrophy - Overview
    • Pipeline Products for Myotonic Dystrophy - Comparative Analysis
  • Myotonic Dystrophy - Therapeutics under Development by Companies
  • Myotonic Dystrophy - Therapeutics under Investigation by Universities/Institutes
  • Myotonic Dystrophy - Pipeline Products Glance
    • Clinical Stage Products
    • Early Stage Products
  • Myotonic Dystrophy - Products under Development by Companies
  • Myotonic Dystrophy - Products under Investigation by Universities/Institutes
  • Myotonic Dystrophy - Companies Involved in Therapeutics Development
    • AMO Pharma Limited
    • BioMarin Pharmaceutical Inc.
    • F. Hoffmann-La Roche Ltd.
    • Ionis Pharmaceuticals, Inc.
    • Marina Biotech, Inc.
    • Sarepta Therapeutics, Inc.
    • WAVE Life Sciences Ltd.
  • Myotonic Dystrophy - Therapeutics Assessment
    • Assessment by Monotherapy Products
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Drug Profiles
    • Antisense Oligonucleotide to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Antisense RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • IONISDMPK-2.5Rx - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • IUCT-169 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • IUCT-290 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • IUCT-309 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • LR-08 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • mexiletine hydrochloride - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PRO-135 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RP-33 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules for Myotonic Dystrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Inhibit MBNL1 for Myotonic Dystrophy Type I - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SRT-149 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SRT-152 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • tideglusib - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • VAL-0411 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Myotonic Dystrophy - Dormant Projects
  • Myotonic Dystrophy - Discontinued Products
  • Myotonic Dystrophy - Product Development Milestones
    • Featured News & Press Releases
      • Sep 19, 2013: AmorChem invests in the development of a therapeutic to treat myotonic dystrophy
      • Dec 20, 2012: Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Myotonic Dystrophy, H2 2016
  • Number of Products under Development for Myotonic Dystrophy - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Development, H2 2016
  • Products under Development by Companies, H2 2016
  • Products under Investigation by Universities/Institutes, H2 2016
  • Myotonic Dystrophy - Pipeline by AMO Pharma Limited, H2 2016
  • Myotonic Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H2 2016
  • Myotonic Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H2 2016
  • Myotonic Dystrophy - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016
  • Myotonic Dystrophy - Pipeline by Marina Biotech, Inc., H2 2016
  • Myotonic Dystrophy - Pipeline by Sarepta Therapeutics, Inc., H2 2016
  • Myotonic Dystrophy - Pipeline by WAVE Life Sciences Ltd., H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Stage and Target, H2 2016
  • Number of Products by Stage and Mechanism of Action, H2 2016
  • Number of Products by Stage and Route of Administration, H2 2016
  • Number of Products by Stage and Molecule Type, H2 2016
  • Myotonic Dystrophy - Dormant Projects, H2 2016
  • Myotonic Dystrophy - Discontinued Products, H2 2016

List of Figures

  • Number of Products under Development for Myotonic Dystrophy, H2 2016
  • Number of Products under Development for Myotonic Dystrophy - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Products, H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Targets, H2 2016
  • Number of Products by Stage and Targets, H2 2016
  • Number of Products by Mechanism of Actions, H2 2016
  • Number of Products by Stage and Mechanism of Actions, H2 2016
  • Number of Products by Routes of Administration, H2 2016
  • Number of Products by Stage and Routes of Administration, H2 2016
  • Number of Products by Molecule Types, H2 2016
  • Number of Products by Stage and Molecule Types, H2 2016
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