PRN: Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review 2017 - 108 Products in Development for DMD, by 57 Companies and Nine Academic Institutions - Research and Markets

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Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review 2017 - 108 Products in Development for DMD, by 57 Companies and Nine Academic Institutions - Research and Markets


DUBLIN, May 29, 2017 /PRNewswire/ --

Research and Markets has announced the addition of the "Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review, 2017" report to their offering.


This report provides an overview of the genetic disorders pipeline landscape. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for Becker and Duchenne Muscular Dystrophy (BMD and DMD) and features dormant and discontinued projects. Both indications covered are inherited muscular dystrophies, a group of genetic, degenerative diseases primarily affecting voluntary muscles.

BMD, the first type of muscular dystrophy, leads to symptoms include pain and sensation, difficulty with running, hopping, and jumping, toe walking, breathing problems, cognitive problems and frequent falls. There are a total of six products in development for this indication, by six seperate companies; the companies operating in this pipeline space are all small pharmaceutical enterprises, and consist of Armgo Pharma, Cardero Therapeutics, Milo Biotechnology, PTC Therapeutics, ReveraGen BioPharma and Sarepta Therapeutics.

DMD is a condition which causes muscle weakness, and is an X-linked disorder. The gene for DMD is present on the X chromosome. It codes for a protein named dystrophin. Dystrophin is essential for providing structural support inside muscle cells. Symptoms usually appear before age 6 and may appear as early as infancy. They include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking.

There is a far larger pipeline for DMD than for BMD; there are a total of 108 products in development for DMD, by 57 companies and nine academic institutions. Key companies operating in this pipeline space include Sarepta Therapeutics, PTC Therapeutics, WAVE Life Sciences, Solid Biosciences, Summit Therapeutics and Catabasis Pharmaceuticals. Of particular importance are Sarepta, who are developing twelve distinct pipeline products for DMD. As with BMD, these are all smaller enterprises, although there is some involvement from larger pharmaceutical companies such as Pfizer and Merck in the DMD pipeline.

The majority of pipeline products for DMD target dystrophin which, as mentioned, is the causative gene for the disease. However, a number of products act on other molecular targets such as growth differentiation factor 8, nuclear factor kappa B and utrophin. The first two of these make up the pipeline for BMD, along with forkhead box protein 1B.


- Which companies are the most active within the pipeline for genetic disorder therapeutics?

- Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?

- To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?

- What are the most important R&D milestones and data publications to have happened in the field of genetic disorder therapeutics?

Key Topics Covered:

1. Report Guidance

2. Executive Summary

3. Introduction

4. Genetic Disorder Cluster Muscular Dystrophy Report Coverage

5. Therapeutics Development

6. Therapeutics Assessment

7. Companies Involved in Therapeutics Development

8. Discontinued Products

9. Product Development Milestones

10. Appendix

Companies Mentioned

  • Acceleron Pharma Inc
  • Akashi Therapeutics Inc
  • Asklepios BioPharmaceutical Inc
  • BioMarin Pharmaceutical Inc
  • Bristol-Myers Squibb Company
  • CRISPR Therapeutics
  • Catabasis Pharmaceuticals Inc
  • Daiichi Sankyo Company Ltd
  • Debiopharm International SA
  • Editas Medicine Inc
  • GW Pharmaceuticals Plc
  • Galapagos NV
  • Genethon SA
  • Idera Pharmaceuticals Inc
  • Italfarmaco SpA
  • La Jolla Pharmaceutical Company
  • Lexicon Pharmaceuticals Inc
  • Merck KGaA
  • Mitobridge Inc
  • Mitochon Pharmaceuticals Inc
  • MyoTherix Inc
  • NicOx SA
  • Nippon Shinyaku Co Ltd
  • Nobelpharma Co Ltd
  • Novartis AG
  • Pfizer Inc
  • Pluristem Therapeutics Inc
  • Prothelia Inc
  • ReveraGen BioPharma Inc
  • Santhera Pharmaceuticals Holding AG
  • Sarepta Therapeutics Inc
  • Strykagen Corp
  • Summit Therapeutics Plc
  • WAVE Life Sciences Ltd

For more information about this report visit

Media Contact:

Laura Wood, Senior Manager

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