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Summary
Global Markets Direct’s, Neurofibromatoses - Pipeline Review, H1 2012, provides an
overview of the Neurofibromatoses therapeutic pipeline. This report provides
information on the therapeutic development for Neurofibromatoses, complete with
latest updates, and special features on late-stage and discontinued projects. It also
reviews key players involved in the therapeutic development for
Neurofibromatoses. Neurofibromatoses - Pipeline Review, H1 2012 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.
Note*: Certain sections in the report may be removed or altered based on the
availability and relevance of data for the indicated disease.
Scope
- A snapshot of the global therapeutic scenario for Neurofibromatoses.
- A review of the Neurofibromatoses products under development by companies
and universities/research institutes based on information derived from company
and industry-specific sources.
- Coverage of products based on various stages of development ranging from
discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined

2. therapeutics.
Table of contents:
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Neurofibromatoses Overview 8
Therapeutics Development 9
An Overview of Pipeline Products for Neurofibromatoses 9
Neurofibromatoses Therapeutics under Development by Companies 11
Neurofibromatoses Therapeutics under Investigation by Universities/Institutes 12
Mid Clinical Stage Products 14
Comparative Analysis 14
Early Clinical Stage Products 15
Comparative Analysis 15
Pre-Clinical Stage Products 16
Comparative Analysis 16
Neurofibromatoses Therapeutics – Products under Development by Companies 17
Neurofibromatoses Therapeutics – Products under Investigation by
Universities/Institutes 18
Companies Involved in Neurofibromatoses Therapeutics Development 19
Arno Therapeutics, Inc. 19
PTC Therapeutics, Inc. 20
Nexgenix Pharmaceuticals, LLC 21
Neurofibromatoses – Therapeutics Assessment 22
Assessment by Monotherapy Products 22
Assessment by Combination Products 23
Assessment by Route of Administration 24
Assessment by Molecule Type 26
Drug Profiles 28
AR-42 - Drug Profile 28
Product Description 28
Mechanism of Action 28
R&D Progress 28

3. PTC299 - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
Rapamycin - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
NXD30001 - Drug Profile 33
Product Description 33
List of Figures
List of Tables are also include.
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