Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Pipeline Therapeutics Development Review H2 2015

Press Release   •   Jan 19, 2016 07:54 EST

This report provides comprehensive information on the therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C and special features on late-stage and discontinued projects.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Complete report on H2, 2015 Pipeline Review of “Acid Sphingomyelinase Deficiency” addition with 20 market data tables and 15 figures, spread across 59 pages is .

This 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

Companies Discussed/Mentioned in this Research:

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

Drugs Profile Discussed in this Research:

arimoclomol, Bryostatin-1, Delta-tocopherol, HPGCD, LJPC-0712, ML-SA1, OKL-1014, OR-0005, Small Molecules for Niemann-Pick Type C Disease, Trappsol Cyclo, vorinostat and VTS-270

Inquire before buying for this report: . (This is a premium report price at US$2000 for a single user PDF license)


  • The report provides a snapshot of the global therapeutic landscape of Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C
  • The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in the therapeutics development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C and enlists all their major and minor projects
  • The report summarizes all the dormant and discontinued pipeline projects
  • A review of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • A detailed assessment of monotherapy and combination therapy pipeline projects
  • Coverage of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C pipeline on the basis of target, MoA, route of administration and molecule type
  • Latest news and deals relating related to pipeline products

Reasons to buy:

  • Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Identify and understand important and diverse types of therapeutics under development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C
  • Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
  • Devise corrective measures for pipeline projects by understanding Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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

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