Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2016 - Global Markets Direct - Market Research

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

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2016 - Global Markets Direct - Market Research
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2016
Published Oct 12, 2016
72 pages — Published Oct 12, 2016
Price US$ 2,000.00  |  Buy this Report Now

About This Report

  
Abstract:

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 Directs 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 pro

  
Source:
Document ID
GMDHC8536IDB
Industry
Format:
PDF Adobe Acrobat
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Sections

TitleStarting PageNumber of Pages
Table of Contents24
  List of Tables51
  List of Figures51
Introduction61
  Global Markets Direct Report Coverage61
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Overview71
Therapeutics Development82
  Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Overview81
  Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Comparative Analysis91
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics under Development by Companies101
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics under Investigation by Universities/Institutes111
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Pipeline Products Glance123
  Late Stage Products121
  Clinical Stage Products131
  Early Stage Products141
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Products under Development by Companies151
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Products under Investigation by Universities/Institutes161
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Companies Involved in Therapeutics Development175
  CTD Holdings, Inc.171
  La Jolla Pharmaceutical Company181
  Merck &Co., Inc.191
  Okklo Life Sciences BV201
  Orphazyme ApS211
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics Assessment229
  Assessment by Monotherapy Products221
  Assessment by Target232
  Assessment by Mechanism of Action252
  Assessment by Route of Administration272
  Assessment by Molecule Type292
Drug Profiles3127
  Antibodies for Metabolic Disorders Drug Profile311
  arimoclomol Drug Profile322
  Bryostatin-1 Drug Profile345
  Delta-tocopherol Drug Profile391
  HPGCD Drug Profile401
  LJPC-0712 Drug Profile411
  MLSA-1 Drug Profile421
  OKL-1014 Drug Profile431
  OR-0005 Drug Profile441
  PERL-101 Drug Profile451
  Small Molecule for Niemann-Pick Type C Disease Drug Profile461
  Small Molecules for Niemann-Pick Type C Disease Drug Profile471
  Small Molecules to Target Cholesterol for Niemann-Pick Type C Disease Drug Profile481
  Trappsol Cyclo Drug Profile493
  vorinostat Drug Profile524
  VTS-270 Drug Profile562
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Dormant Projects581
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Product Development Milestones5912
  Featured News &Press Releases591
    Sep 15, 2016: CTD Holdings Announces MHRA Approval of Clinical Trial Application for Treatment of NPC With Trappsol Cyclo591
    Sep 12, 2016: CTD Holdings Files Clinical Trial Application With Swedish Medical Products Agency to Continue Advancing Its Drug Development Program601
    Sep 06, 2016: CTD Holdings Announces FDA Acceptance of Investigational New Drug Application for Treatment of NPC With Trappsol Cyclo601
    Aug 16, 2016: CTD Holdings Meets With Parent Advocate Groups and Physicians611
    Aug 08, 2016: CTD Holdings Files Investigational New Drug Application With US FDA to Further Advance Its Drug Development Program621
    Aug 01, 2016: CTD Holdings Files Clinical Trial Application with U.K. Regulatory Agency to Advance Its Drug Development Program631
    Jun 28, 2016: CTD Holdings Announces Joint Presentation With UCSF Benioff Children Hospital Oakland on Patient Experience in Compassionate Treatment Program641
    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 Conference641
    Feb 29, 2016: CTD Holdings Clinical Program to Move Forward in the U.S.651
    Feb 18, 2016: Parent Advocates Join CTD Holdings Family and Physicians Listening Circle on NPC Treatment661
    Dec 14, 2015: CTD Holdings' Clinical Trial Design Receives Positive Responses From U.K.'s Medicines and Healthcare Products Regulatory Agency661
    Sep 16, 2015: N. Scott Fine Named CEO of CTD Holdings671
    Jun 24, 2015: Neurotrope to Conduct Study on Niemann-Pick Type C, a Devastating Rare Pediatric Disease681
    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 C691
    Jan 27, 2015: FDA Accepts CTD Holdings Drug Master File for Second-Generation Orphan Drug701
Appendix712
  Methodology711
  Coverage711
  Secondary Research711
  Primary Research711
  Expert Panel Validation711
  Contact Us711
  Disclaimer721

Table Of Contents

Global Markets Direct - Market Research—Global Markets Direct is a leading provider of global business intelligence and market analysis. It publishes a range of high quality market reports created by its large research and analysis capability and drawing from its extensive unique databases of industry-specific information.

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Cite this Report

  
MLA:
Global Markets Direct - Market Research. "Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2016" Oct 12, 2016. Alacra Store. May 05, 2025. <http://www.alacrastore.com/storecontent/Global-Markets-Direct-Market-Research/Acid-Sphingomyelinase-Deficiency-Niemann-Pick-Disease-Type-C-Pipeline-Review-H2-2016-2088-16664>
  
APA:
Global Markets Direct - Market Research. (2016). Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2016 Oct 12, 2016. New York, NY: Alacra Store. Retrieved May 05, 2025 from <http://www.alacrastore.com/storecontent/Global-Markets-Direct-Market-Research/Acid-Sphingomyelinase-Deficiency-Niemann-Pick-Disease-Type-C-Pipeline-Review-H2-2016-2088-16664>
  
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