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筋緊張性ジストロフィー:パイプライン分析

Myotonic Dystrophy - Pipeline Review, H1 2016

発行 Global Markets Direct 商品コード 271823
出版日 ページ情報 英文 63 Pages
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筋緊張性ジストロフィー:パイプライン分析 Myotonic Dystrophy - Pipeline Review, H1 2016
出版日: 2016年02月24日 ページ情報: 英文 63 Pages
概要

筋緊張性ジストロフィーとは遺伝性の筋ジストロフィーの一種で、筋肉や人体全体に悪影響を及ぼします。主な症状としては、呼吸困難や嚥下障害、消化障害、日中の過度の眠気、学習障害などが挙げられます。主な治療法としては、外科手術やコルチコステロイド剤などがあります。

当レポートでは、世界各国での筋緊張性ジストロフィー治療用のパイプライン製品の開発状況について分析し、製品開発・上市の最新動向や、治験の各段階にある製品の一覧、主要企業のプロファイル、主な薬剤の概要、最新の業界動向などの情報をお届けします。

目次

イントロダクション

  • 分析範囲

筋緊張性ジストロフィーの概要

治療薬の開発

  • 筋緊張性ジストロフィー向けパイプライン製品:概要
  • 筋緊張性ジストロフィー向けパイプライン製品:比較分析

各企業で開発中の筋緊張性ジストロフィー治療薬

大学/研究機関で研究中の筋緊張性ジストロフィー治療薬

パイプライン製品の概略

  • 治験段階の製品
  • 初期段階の製品

筋緊張性ジストロフィー治療薬:開発中の製品の一覧(企業別)

筋緊張性ジストロフィー治療薬:研究中の製品の一覧(大学/研究機関別)

筋緊張性ジストロフィー治療薬の開発に従事している企業

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

筋緊張性ジストロフィー:治療薬の評価

  • 単剤治療薬の場合
  • 標的別
  • 作用機序別
  • 投与方法別
  • 分子の種類別

薬剤のプロファイル

  • 脊髄性筋萎縮症・筋緊張性ジストロフィー向けアンチセンスオリゴヌクレオチド
    • 製品概要
    • 機能メカニズム
    • 研究開発(R&D)の進展状況
  • ISIS-DMPKRx
  • PRO-135
  • 筋緊張性ジストロフィー向けRNA干渉オリゴヌクレオチド
  • CUG RNAを標的とした筋緊張性ジストロフィー1型向け医薬品
  • RNAを標的とした筋緊張性ジストロフィー向け小分子
  • 筋緊張性ジストロフィー1型向け医薬品
  • RNAを標的とした筋緊張性ジストロフィー向け小分子
  • 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

筋緊張性ジストロフィー治療薬:パイプライン製品の最新動向

筋緊張性ジストロフィー関連製品の開発のマイルストーン

  • 注目すべき最新動向・プレスリリース

付録

図表一覧

このページに掲載されている内容は最新版と異なる場合があります。詳細はお問い合わせください。

目次
Product Code: GMDHC7657IDB

Summary

Global Markets Direct's, 'Myotonic Dystrophy - Pipeline Review, H1 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.
    • Valentia Biopharma S.L.
    • 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
    • AMO-02 - 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
    • IONIS-DMPK2.5Rx - 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
    • 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 Type 1 - 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
    • VAL-0411 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-001 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-002 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-003 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-004 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-005 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-014 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-015 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-016 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-017 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • VLT-025 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
  • Myotonic Dystrophy - Recent Pipeline Updates
  • Myotonic Dystrophy - Dormant Projects
  • Myotonic Dystrophy - Product Development Milestones
    • Featured News & Press Releases
      • Feb 25, 2014: VLT Biopharma gets the Orphan Drug designation for VLT015
      • 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, H1 2016
  • Number of Products under Development for Myotonic Dystrophy - Comparative Analysis, H1 2016
  • Number of Products under Development by Companies, H1 2016
  • Number of Products under Investigation by Universities/Institutes, H1 2016
  • Comparative Analysis by Clinical Stage Development, H1 2016
  • Comparative Analysis by Early Stage Development, H1 2016
  • Products under Development by Companies, H1 2016
  • Products under Investigation by Universities/Institutes, H1 2016
  • Myotonic Dystrophy - Pipeline by AMO Pharma Limited, H1 2016
  • Myotonic Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H1 2016
  • Myotonic Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016
  • Myotonic Dystrophy - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016
  • Myotonic Dystrophy - Pipeline by Marina Biotech, Inc., H1 2016
  • Myotonic Dystrophy - Pipeline by Valentia Biopharma S.L., H1 2016
  • Myotonic Dystrophy - Pipeline by WAVE Life Sciences Ltd., H1 2016
  • Assessment by Monotherapy Products, H1 2016
  • Number of Products by Stage and Target, H1 2016
  • Number of Products by Stage and Mechanism of Action, H1 2016
  • Number of Products by Stage and Route of Administration, H1 2016
  • Number of Products by Stage and Molecule Type, H1 2016
  • Myotonic Dystrophy Therapeutics - Recent Pipeline Updates, H1 2016
  • Myotonic Dystrophy - Dormant Projects, H1 2016

List of Figures

  • Number of Products under Development for Myotonic Dystrophy, H1 2016
  • Number of Products under Development for Myotonic Dystrophy - Comparative Analysis, H1 2016
  • Number of Products under Development by Companies, H1 2016
  • Comparative Analysis by Early Stage Products, H1 2016
  • Assessment by Monotherapy Products, H1 2016
  • Number of Products by Targets, H1 2016
  • Number of Products by Stage and Targets, H1 2016
  • Number of Products by Mechanism of Actions, H1 2016
  • Number of Products by Stage and Mechanism of Actions, H1 2016
  • Number of Products by Routes of Administration, H1 2016
  • Number of Products by Stage and Routes of Administration, H1 2016
  • Number of Products by Molecule Types, H1 2016
  • Number of Products by Stage and Molecule Types, H1 2016
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