Muscular Dystrophy - Pipeline Review, Half Year is built using data and information sourced from 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.

1. Muscular Dystrophy - Pipeline Review, H1 2013
Global Markets Direct’s, 'Muscular Dystrophy - Pipeline Review, H1 2013', provides an overview of
the indication’s therapeutic pipeline. This report provides information on the therapeutic development
for Muscular Dystrophy, complete with latest updates, and special features on late-stage and discontinued
projects. It also reviews key players involved in the therapeutic development for Muscular Dystrophy.
Muscular Dystrophy - Pipeline Review, Half Year 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 Muscular Dystrophy. A review of the Muscular Dystrophy
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 Muscular Dystrophy pipeline on the basis of route of administration
and molecule type. 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 Muscular
Dystrophy. 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 of the most promising pipeline. Devise corrective measures for pipeline projects by understanding
Muscular Dystrophy 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.
table Of Content
introduction
global Markets Direct Report Coverage
muscular Dystrophy Overview
therapeutics Development
an Overview Of Pipeline Products For Muscular Dystrophy
muscular Dystrophy Therapeutics Under Development By Companies
muscular Dystrophy Therapeutics Under Investigation By Universities/institutes
mid Clinical Stage Products
Muscular Dystrophy - Pipeline Review, H1 2013

2. comparative Analysis
early Clinical Stage Products
comparative Analysis
discovery And Pre-clinical Stage Products
comparative Analysis
muscular Dystrophy Therapeutics – Products Under Development By Companies
muscular Dystrophy Therapeutics – Products Under Investigation By Universities/institutes
companies Involved In Muscular Dystrophy Therapeutics Development
genzyme Corporation
mdrna, Inc.
pfizer Inc.
benitec Ltd.
calzada Limited
nutra Pharma Corporation
santhera Pharmaceuticals Holding Ag
scynexis, Inc.
fate Therapeutics, Inc.
sanbio, Inc.
prosensa Therapeutics B.v.
muscular Dystrophy – Therapeutics Assessment
assessment By Monotherapy Products
assessment By Route Of Administration
assessment By Molecule Type
drug Profiles
aod-9604 - Drug Profile
product Description
mechanism Of Action
r&d Progress
omigapil - Drug Profile
product Description
mechanism Of Action
r&d Progress
sb-308 - Drug Profile
product Description
mechanism Of Action
r&d Progress
ligand-1 - Drug Profile
product Description
mechanism Of Action
r&d Progress
pro-135 - Drug Profile
product Description
mechanism Of Action
Muscular Dystrophy - Pipeline Review, H1 2013

3. r&d Progress
raav1.tmck.human-alpha-sarcoglycan - Drug Profile
product Description
mechanism Of Action
r&d Progress
gamma-sarcoglycan Gene Therapy - Drug Profile
product Description
mechanism Of Action
r&d Progress
gamma-sarcoglycan Gene Therapy - Drug Profile
product Description
mechanism Of Action
r&d Progress
ft-301 - Drug Profile
product Description
mechanism Of Action
r&d Progress
prt-01 - Drug Profile
product Description
mechanism Of Action
r&d Progress
prt-300 - Drug Profile
product Description
mechanism Of Action
r&d Progress
wnt7a--based Protein Thearpeutics - Drug Profile
product Description
mechanism Of Action
r&d Progress
pf-06252616 - Drug Profile
product Description
mechanism Of Action
r&d Progress
ddrnai Program For Oculopharyngeal Muscular Dystrophy - Drug Profile
product Description
mechanism Of Action
r&d Progress
aav5 Gene Therapy - Drug Profile
product Description
mechanism Of Action
r&d Progress
sirolimus - Drug Profile
product Description
Muscular Dystrophy - Pipeline Review, H1 2013

4. mechanism Of Action
r&d Progress
cyclophilin Inhibitory Compounds - Drug Profile
product Description
mechanism Of Action
r&d Progress
rnase H-active Antisense Oligos - Drug Profile
product Description
mechanism Of Action
r&d Progress
vlt-001 - Drug Profile
product Description
mechanism Of Action
r&d Progress
vlt-002 - Drug Profile
product Description
mechanism Of Action
r&d Progress
drug For Myotonic Dystrophy - Drug Profile
product Description
mechanism Of Action
r&d Progress
drug For Myotonic Dystrophy - Drug Profile
product Description
mechanism Of Action
r&d Progress
val-0411 - Drug Profile
product Description
mechanism Of Action
r&d Progress
muscular Dystrophy Program - Drug Profile
product Description
mechanism Of Action
r&d Progress
gsmtx-4 - Drug Profile
product Description
mechanism Of Action
r&d Progress
srt-149 - Drug Profile
product Description
mechanism Of Action
r&d Progress
srt-152 - Drug Profile
Muscular Dystrophy - Pipeline Review, H1 2013

5. product Description
mechanism Of Action
r&d Progress
val-1205 - Drug Profile
product Description
mechanism Of Action
r&d Progress
muscular Dystrophy Therapeutics – Drug Profile Updates
muscular Dystrophy Therapeutics – Discontinued Products
muscular Dystrophy Therapeutics - Dormant Products
muscular Dystrophy – Product Development Milestones
featured News & Press Releases
dec 03, 2012: Benitec Biopharma’s Muscular Dystrophy Program Demonstrates Significant Gene
Silencing
apr 11, 2012: Santhera Pharma Joins Eu-funded Endostem Consortium In Preparation For Clinical Study
With Omigapil In Congenital Muscular Dystrophies
dec 14, 2011: Calzada Announces New Applications For Aod9604
sep 23, 2011: Prothelia's Laminin-111 Receives Orphan Drug Designation For Treatment Of Congenital
Muscular Dystrophy
feb 08, 2011: Santhera Obtains Us And Canadian Patents For Use Of Omigapil For Treatment Of Congenital
Muscular Dystrophy
dec 07, 2010: Santhera Wins European Patent For Use Of Omigapil In Treatment Of Congenital Muscular
Dystrophy
oct 31, 2006: Avanir Receives Approvable Letter From Fda For Zenvia.
appendix
methodology
coverage
secondary Research
primary Research
expert Panel Validation
contact Us
disclaimer
ResearchMoz(http://www.researchmoz.us/) is the one stop online destination to find and buy market research
reports & Industry Analysis. We fulfill all your research needs spanning across industry verticals with our
huge collection of market research reports. We provide our services to all sizes of organizations and across all
industry verticals and markets. Our Research Coordinators have in-depth knowledge of reports as well as
publishers and will assist you in making an informed decision by giving you unbiased and deep insights on
which reports will satisfy your needs at the best price.
Contact:
Muscular Dystrophy - Pipeline Review, H1 2013

6. M/s Sheela,
90 State Street,
Suite 700,
Albany NY - 12207
United States
Tel: +1-518-618-1030
USA - Canada Toll Free 866-997-4948
Email: sales@researchmoz.us
Website: http://www.researchmoz.us/
Muscular Dystrophy - Pipeline Review, H1 2013