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第三型黏多糖症(MPSIII)(聖菲利柏氏症):開發平台分析

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2016

出版商 Global Markets Direct 商品編碼 257789
出版日期 內容資訊 英文 75 Pages
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第三型黏多糖症(MPSIII)(聖菲利柏氏症):開發平台分析 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2016
出版日期: 2016年06月08日 內容資訊: 英文 75 Pages
簡介

第三型黏多糖症(MPSIII)是以聖菲利柏氏症知名的遺傳性代謝疾病,是體內欠缺可分解葡萄糖胺聚糖 (glycosaminoglycans, GAG)分子所需的某種特定酵素或是功能不全造成的疾病。常見的症狀有中風、多動、肝臟或脾臟肥大、嚴重下痢或便祕、扁桃腺或咽喉扁桃腺肥大等。治療方法則包含了酵素補充療法(ERT)等。

本報告提供第三型黏多糖症(MPSIII)(聖菲利柏氏症)的治療藥開發情形調查分析,提供您開發中產品概要,臨床實驗的各階段產品概要,主要企業簡介,藥物簡介,開發中產品的最新趨勢,最新消息和新聞稿等相關的系統性資訊。

目錄

簡介

第三型黏多糖症(MPSIII)(聖菲利柏氏症)概要

治療藥的開發

  • 開發中產品:概要
  • 開發中產品:比較分析

開發中的治療藥:各企業

調查中的治療藥:各大學/研究機關

開發中產品的概要

  • 臨床實驗階段的產品
  • 初期階段的產品

開發中的產品:各企業

調查中的產品:各大學/研究機關

開發治療藥的企業

  • Abeona Therapeutics, Inc.
  • Alexion Pharmaceuticals, Inc.
  • ArmaGen Inc.
  • Axcentua Pharmaceuticals AB
  • BioMarin Pharmaceutical Inc.
  • Dorphan S.A.
  • Laboratorios Del Dr. Esteve S.A.
  • Lysogene
  • Shire Plc
  • Swedish Orphan Biovitrum AB
  • UniQure N.V.

治療藥的評估

  • 單獨療法的情況
  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

開發中產品的最新趨勢

暫停的計劃

產品開發的里程碑

  • 最新消息和新聞稿

附錄

圖表

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
Product Code: GMDHC8188IDB

Summary

Global Markets Direct's, 'Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2016', provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome )
  • The report reviews pipeline therapeutics for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) therapeutics and enlists all their major and minor projects
  • The report assesses Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome )

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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome )
  • 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) 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
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Overview
  • Therapeutics Development
    • Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Overview
    • Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Development by Companies
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Investigation by Universities/Institutes
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Products Glance
    • Clinical Stage Products
    • Early Stage Products
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Development by Companies
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Investigation by Universities/Institutes
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Companies Involved in Therapeutics Development
    • Abeona Therapeutics, Inc.
    • Alexion Pharmaceuticals, Inc.
    • ArmaGen Inc.
    • Axcentua Pharmaceuticals AB
    • BioMarin Pharmaceutical Inc.
    • Dorphan S.A.
    • Laboratorios Del Dr. Esteve S.A.
    • Lysogene
    • Shire Plc
    • Swedish Orphan Biovitrum AB
    • UniQure N.V.
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - 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
    • ABO-101 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • ABO-102 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • AGT-184 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • AMT-110 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • AXP-10711 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • BMN-250 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • EGT-101 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • EGT-201 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • LYSSAF-301 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • LYSSAF-302 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Proteins for Sanfilippo Syndrome - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • SBC-103 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • SHP-610 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Small Molecule for Sanfilippo Syndrome - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • SOBI-003 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Stem Cell Therapy for Mucopolysaccharidosis Type IIIA - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Stem Cell Therapy to Activate NAG for Mucopolysaccharidosis III - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Recent Pipeline Updates
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Product Development Milestones
    • Featured News & Press Releases
      • May 24, 2016: Abeona Therapeutics Announces FDA Allowance of Investigational New Drug for Phase 1/2 Clinical Study With ABO-101 Gene Therapy for Patients With Sanfilippo Syndrome Type B (MPS IIIB)
      • Feb 25, 2016: ESTEVE Provides Update On EGT-101 For Sanfilippo A Syndrome
      • Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome
      • Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome
      • May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA
      • Apr 02, 2014: Orphan designation granted for MPSIII
      • Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease
      • Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1 2016
  • Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - 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
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Abeona Therapeutics, Inc. , H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Alexion Pharmaceuticals, Inc., H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc., H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Axcentua Pharmaceuticals AB, H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by BioMarin Pharmaceutical Inc., H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Dorphan S.A., H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Laboratorios Del Dr. Esteve S.A., H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lysogene, H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Shire Plc, H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by UniQure N.V., 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
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Therapeutics - Recent Pipeline Updates, H1 2016
  • Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, H1 2016

List of Figures

  • Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1 2016
  • Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H1 2016
  • Number of Products under Development by Companies, H1 2016
  • Comparative Analysis by Clinical Stage Development, 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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