Market Overview

Gene Therapy for Muscular Dystrophy | A Drug Pipeline Analysis Report 2018 | Technavio


Technavio has announced their latest drug pipeline analysis report on gene
therapy for muscular dystrophy
. The report includes a detailed
analysis of the pipeline molecules under investigation within the
defined data collection period to treat muscular dystrophy.

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Technavio has published a new report on the gene therapy pipeline for muscular dystrophy, including  ...

Technavio has published a new report on the gene therapy pipeline for muscular dystrophy, including a detailed study of the pipeline molecules. (Graphic: Business Wire)

This report by Technavio
presents a detailed analysis of the market, including regulatory
framework, drug development strategies, recruitment strategies, and key
companies that are expected to play an essential role in the growth of
the market.

This report is available at a USD 1,000 discount for a limited time
market snapshot before purchasing

Muscular dystrophy: Market overview

A muscular dystrophy is a group of muscle disease that causes an
increasing weakening and breakdown of degeneration of skeletal muscles.
The individuals suffering from muscular dystrophy start losing muscle
mass. The most common cause of muscular dystrophy is the mutation of the
protein dystrophin. It is also caused by the genetic mutations that
interfere with the muscle protein production. Factors such as the site
of affected muscles, the degree of weakness, progression speed, and the
first sign of symptoms will decide the type of muscular dystrophy.

According to a senior market research analyst at Technavio, "Dystrophin
is a part of complex group of proteins that enable muscles to work
correctly. There are over 30 various types of dystrophies with a few
being the most common types such as Duchenne muscular dystrophy, Becker
muscular dystrophy, and limb-girdle muscular dystrophy. Duchenne
muscular dystrophy occurs mostly in boys and very rarely in girls.
According to various studies, the average life expectancy of patients
with Duchenne muscular dystrophy is around 26 years, while the life
expectancy of other types of muscular dystrophies is slightly higher."

Muscular dystrophy: Segmentation analysis

This pipeline analysis report segments the muscular dystrophy market
based on therapies employed (monotherapy), RoA (intravenous and
parenteral), therapeutic modality (gene and oligonucleotides), targets
(micro-dystrophin, exon 51, exon 53, beta-sarcoglycan,
alpha-sarcoglycan, dysferlin, anoctamin 5, gamma-sarcoglycan, and
mini-dystrophin), MoA (gene therapy and antisense gene therapy),
geographical segmentation (US, UK, and France) and recruitment status
(active, not recruiting and recruiting). It provides an in-depth
analysis of the prominent factors influencing the market, including
drivers, opportunities, trends, and industry-specific challenges.

Based on the mechanism of action, around 88% of the molecules that are
being investigated for the treatment of muscular dystrophy are gene

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Some of the key topics covered in the report include:

Scope of the Report

Regulatory Framework

Drug Development Landscape

  • Drugs under development
  • Indications coverage

Drug Development Strategies

  • Therapies employed
  • RoA
  • Therapeutic modality
  • Geographical coverage

Recruitment Strategies

  • Recruitment status
  • Gender
  • Age

Key Companies

  • Type of players
  • Company overview

Discontinued or Dormant Molecules

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