Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2012

Global Markets Direct, April 2012, Pages: 62

Spinal Muscular Atrophy (SMA) – Pipeline Review, H1 2012

Summary

Global Markets Direct’s, 'Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2012', provides an overview of the Spinal Muscular Atrophy (SMA) therapeutic pipeline. This report provides information on the therapeutic development for Spinal Muscular Atrophy (SMA) , complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Spinal Muscular Atrophy (SMA) . 'Spinal Muscular Atrophy (SMA) - 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 Spinal Muscular Atrophy (SMA) .
- A review of the Spinal Muscular Atrophy (SMA) 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 therapeutics.
- Coverage of the Spinal Muscular Atrophy (SMA) pipeline on the basis of route of administration and molecule type.
- Profiles of late-stage pipeline products featuring sections on product description, mechanism of action and research & development progress.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.

Reasons to buy

- Identify and understand important and diverse types of therapeutics under development for Spinal Muscular Atrophy (SMA) .
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players with the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Spinal Muscular Atrophy (SMA) pipeline depth and focus of Spinal Muscular Atrophy (SMA) 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.

Keywords

Spinal Muscular Atrophy (SMA) Therapeutic Products under Development, Key Players in Spinal Muscular Atrophy (SMA) Therapeutics, Spinal Muscular Atrophy (SMA) Pipeline Overview, Spinal Muscular Atrophy (SMA) Pipeline, Spinal Muscular Atrophy (SMA) Pipeline Assessment

List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Spinal Muscular Atrophy (SMA) Overview
Therapeutics Development
An Overview of Pipeline Products for Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) Therapeutics under Development by Companies
Spinal Muscular Atrophy (SMA) Therapeutics under Investigation by Universities/Institutes
Mid Clinical Stage Products
Comparative Analysis
Early Clinical Stage Products
Comparative Analysis
Discovery and Pre-Clinical Stage Products
Comparative Analysis
Spinal Muscular Atrophy (SMA) Therapeutics – Products under Development by Companies
Spinal Muscular Atrophy (SMA) Therapeutics – Products under Investigation by Universities/Institutes
Companies Involved in Spinal Muscular Atrophy (SMA) Therapeutics Development
F. Hoffmann-La Roche Ltd.
Isis Pharmaceuticals, Inc.
Oxford BioMedica plc
Repligen Corporation
Trophos SA
Nexgenix Pharmaceuticals, LLC
Spinal Muscular Atrophy (SMA) – Therapeutics Assessment
Assessment by Monotherapy Products
Assessment by Combination Products
Assessment by Route of Administration
Assessment by Molecule Type
Drug Profiles
RG3039 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Olesoxime - Drug Profile
Product Description
Mechanism of Action
R&D Progress
SMN1-G - Drug Profile
Product Description
Mechanism of Action
R&D Progress
NXD30001 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Sodium Phenylbutyrate (NaPB) - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Valproic Acid - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Valproic acid + Levocarnitine - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Somatotropin - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ISIS-SMNRx - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Depakote + Carnitor - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Drug For Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Drug For Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Spinal Muscular Atrophy Programme - Drug Profile
Product Description
Mechanism of Action
R&D Progress
RE-003 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Fasudil - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Spinal Muscular Atrophy (SMA) Therapeutics – Drug Profile Updates
Spinal Muscular Atrophy (SMA) Therapeutics – Discontinued Products
Spinal Muscular Atrophy (SMA) Therapeutics - Dormant Products
Spinal Muscular Atrophy (SMA) – Product Development Milestones
Featured News & Press Releases
Mar 06, 2012: New Drug Shows Promise In Treating Spinal Muscular Atrophy, Researchers Discover
Dec 19, 2011: Isis Initiates Phase I Clinical Study Of ISIS-SMNRx In Patients With Spinal Muscular Atrophy
Oct 28, 2011: Repligen Provides Update On Phase I Trial For Families Of Spinal Muscular Atrophy Drug RG3039
Sep 08, 2011: Trophos Completes Patient Enrolment In Pivotal Efficacy Study Of Olesoxime In Spinal Muscular Atrophy
Jun 23, 2011: Repligen Receives US Fast Track Designation And European Orphan Medicinal Product Recommendation For RG3039 In Spinal Muscular Atrophy
May 19, 2011: Repligen Receives FDA Approval To Initiate Phase I Clinical Trial Of RG3039 For Treatment Of Spinal Muscular Atrophy
Apr 15, 2011: Trophos Presents Data At AAN On Olesoxime In Spinal Muscular Atrophy And MS
Oct 15, 2010: Trophos Initiates Pivotal Efficacy Study Of Olesoxime In Spinal Muscular Atrophy
Mar 12, 2009: Isis Pharmaceuticals Adds New Drug ISIS-SMNRx To Development Pipeline For The Treatment Of Spinal Muscular Atrophy
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products Under Development for Spinal Muscular Atrophy (SMA), H1 2012
Products under Development for Spinal Muscular Atrophy (SMA) – Comparative Analysis, H1 2012
Number of Products under Development by Companies, H1 2012
Number of Products under Investigation by Universities/Institutes, H1 2012
Comparative Analysis by Mid Clinical Stage Development, H1 2012
Comparative Analysis by Early Clinical Stage Development, H1 2012
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H1 2012
Products under Development by Companies, H1 2012
Products under Investigation by Universities/Institutes, H1 2012
F. Hoffmann-La Roche Ltd., H1 2012
Isis Pharmaceuticals, Inc., H1 2012
Oxford BioMedica plc, H1 2012
Repligen Corporation, H1 2012
Trophos SA, H1 2012
Nexgenix Pharmaceuticals, LLC, H1 2012
Assessment by Monotherapy Products, H1 2012
Assessment by Combination Products, H1 2012
Assessment by Stage and Route of Administration, H1 2012
Assessment by Stage and Molecule Type, H1 2012
Spinal Muscular Atrophy (SMA) Therapeutics – Drug Profile Updates
Spinal Muscular Atrophy (SMA) Therapeutics – Discontinued Products
Spinal Muscular Atrophy (SMA) Therapeutics – Dormant Products

List of Figures
Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2012
Products under Development for Spinal Muscular Atrophy (SMA) – Comparative Analysis, H1 2012
Products under Development by Companies, H1 2012
Products under Investigation by Universities/Institutes, H1 2012
Mid Clinical Stage Products, H1 2012
Early Clinical Stage Products, H1 2012
Discovery and Pre-Clinical Stage Products, H1 2012
Assessment by Monotherapy Products, H1 2012
Assessment by Combination Products, H1 2012
Assessment by Route of Administration, H1 2012
Assessment by Stage and Route of Administration, H1 2012
Assessment by Molecule Type, H1 2012
Assessment by Stage and Molecule Type, H1 2012

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