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酸性神經磷脂(酉每)缺乏症(尼曼匹克症)C型:開發平台分析

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2016

出版商 Global Markets Direct 商品編碼 332892
出版日期 內容資訊 英文 72 Pages
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酸性神經磷脂(酉每)缺乏症(尼曼匹克症)C型:開發平台分析 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2016
出版日期: 2016年10月12日 內容資訊: 英文 72 Pages
簡介

酸性神經磷脂(酉每)缺乏症(尼曼匹克症)以溶小體儲積症的1個,是帶來代謝的影響,遺傳基因的突然變異起因於的疾病。被體內的細胞,尤其是網內系細胞(單核吞噬細胞系)sufingo脂質積蓄。C型(NPC)特別與A型和B型(ASMD)很大地不同。患者,不能在膽固醇和細胞內其他脂質中適當代謝。根據那個,被肝臟和脾臟膽固醇,還有腦內脂質過度積蓄了。 症狀,肝臟肥大,腦功能障礙,步行困難,吞嚥障礙,接觸過敏,表達困難,筋緊張的消失,學習障礙等能看。

本報告提供全球酸性神經磷脂(酉每)缺乏症(尼曼匹克症)C型的開發中產品的開發情形相關分析,送交調查了開發中產品的最新趨勢,及臨床實驗的各階段有的產品的一覽,主要企業簡介,主要藥物概要(產品概要,功能機制,研究開發的發展情形),最新的產業趨勢等的結果。

簡介

  • 調查範圍
  • 酸性神經磷脂(酉每)缺乏症 (尼曼匹克症)) C型 概要

治療藥的開發

  • 開發中產品;概要
  • 開發中產品;比較分析

在企業正在開發的治療藥

在大學/機關研究中的治療藥

開發中產品概況

  • 臨床階段的產品
  • 初期階段的產品

在企業開發中的產品

在大學/機關研究中的產品

治療藥開發作的企業

  • La Jolla Pharmaceutical Company
  • Merck & Co., Inc.
  • Okklo Life Sciences BV
  • Orphazyme ApS

治療藥的評估

  • 單劑產品
  • 按標的
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

  • arimoclomol
  • Bryostatin-1
  • Delta-tocopherol
  • LJPC-0712
  • ML-SA1
  • OKL-1014
  • OR-0005
  • vorinostat
  • VTS-270

最近的開發平台趨勢

暫停中的計劃

暫停中的計劃

附錄

圖表

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目錄
Product Code: GMDHC8536IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency Type C - Pipeline Review, H2 2016, provides an overview of the Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) pipeline landscape.

Niemann-Pick C disease is one of a group of lysosomal storage diseases that affect metabolism and that are caused by genetic mutations. It involves the accumulation of sphingolipids in cells throughout the body, particularly reticuloendothelial cells (the mononuclear phagocyte system). Symptoms include enlarged liver, brain damage, difficulty walking and swallowing, increased sensitivity to touch, difficulty speaking, loss of muscle tone, learning difficulties.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Acid Sphingomyelinase Deficiency Type C - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Acid Sphingomyelinase Deficiency Type C (Genetic Disorders), 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 Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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, IND/CTA Filed, Preclinical and Discovery stages are 2, 1, 1, 7 and 1 respectively for Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 2 molecules, respectively for Acid Sphingomyelinase Deficiency Type C.

Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) 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 Acid Sphingomyelinase Deficiency Type C (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) 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 Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) 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 Acid Sphingomyelinase Deficiency Type C (Genetic Disorders)

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 Acid Sphingomyelinase Deficiency Type C (Genetic Disorders).
  • 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 Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) 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

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Overview
  • Therapeutics Development
    • Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Overview
    • Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Comparative Analysis
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics under Development by Companies
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics under Investigation by Universities/Institutes
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Products Glance
    • Late Stage Products
    • Clinical Stage Products
    • Early Stage Products
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Products under Development by Companies
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Products under Investigation by Universities/Institutes
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Companies Involved in Therapeutics Development
    • CTD Holdings, Inc.
    • La Jolla Pharmaceutical Company
    • Merck & Co., Inc.
    • Okklo Life Sciences BV
    • Orphazyme ApS
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - 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
    • Antibodies for Metabolic Disorders - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • arimoclomol - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Bryostatin-1 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Delta-tocopherol - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • HPGCD - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • LJPC-0712 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • MLSA-1 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • OKL-1014 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • OR-0005 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PERL-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecule for Niemann-Pick Type C Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules for Niemann-Pick Type C Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Target Cholesterol for Niemann-Pick Type C Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Trappsol Cyclo - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • vorinostat - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • VTS-270 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones
    • Featured News & Press Releases
      • Sep 15, 2016: CTD Holdings Announces MHRA Approval of Clinical Trial Application for Treatment of NPC With Trappsol Cyclo
      • Sep 12, 2016: CTD Holdings Files Clinical Trial Application With Swedish Medical Products Agency to Continue Advancing Its Drug Development Program
      • Sep 06, 2016: CTD Holdings Announces FDA Acceptance of Investigational New Drug Application for Treatment of NPC With Trappsol Cyclo
      • Aug 16, 2016: CTD Holdings Meets With Parent Advocate Groups and Physicians
      • Aug 08, 2016: CTD Holdings Files Investigational New Drug Application With US FDA to Further Advance Its Drug Development Program
      • Aug 01, 2016: CTD Holdings Files Clinical Trial Application with U.K. Regulatory Agency to Advance Its Drug Development Program
      • Jun 28, 2016: CTD Holdings Announces Joint Presentation With UCSF Benioff Children Hospital Oakland on Patient Experience in Compassionate Treatment Program
      • May 03, 2016: CTD Holdings to Present "Management of Niemann-Pick Type C With Intravenous Hydroxypropyl- b -Cyclodextrin" at Ara Parseghian Medical Research Foundation Annual Conference
      • Feb 29, 2016: CTD Holdings Clinical Program to Move Forward in the U.S.
      • Feb 18, 2016: Parent Advocates Join CTD Holdings Family and Physicians Listening Circle on NPC Treatment
      • Dec 14, 2015: CTD Holdings' Clinical Trial Design Receives Positive Responses From U.K.'s Medicines and Healthcare Products Regulatory Agency
      • Sep 16, 2015: N. Scott Fine Named CEO of CTD Holdings
      • Jun 24, 2015: Neurotrope to Conduct Study on Niemann-Pick Type C, a Devastating Rare Pediatric Disease
      • Jun 12, 2015: Neurotrope to Present Findings of In-vitro Studies of Bryostatin's Effects on Niemann Pick Type C1 Cells at the Annual "Michael, Marcia & Christa Parseghian Scientific Conference" for Niemann-Pick Type C
      • Jan 27, 2015: FDA Accepts CTD Holdings Drug Master File for Second-Generation Orphan Drug
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2016
  • Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - 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 Late Stage Development, 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
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by CTD Holdings, Inc., H2 2016
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by La Jolla Pharmaceutical Company, H2 2016
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Merck & Co., Inc., H2 2016
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Okklo Life Sciences BV, H2 2016
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Orphazyme ApS, 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
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H2 2016

List of Figures

  • Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2016
  • Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - 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 Early Stage Products, H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Top 10 Targets, H2 2016
  • Number of Products by Stage and Top 10 Targets, H2 2016
  • Number of Products by Top 10 Mechanism of Actions, H2 2016
  • Number of Products by Stage and Top 10 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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