Duchenne Muscular Dystrophy (DMD): KOL Insight 2020

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Publication Date:
June 2020
Licence Type:
Multi-user licence
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Targeted Therapies in DMD: KOL Insight

Are the days of steroids being the standard of care for DMD coming to an end? What benefits do KOLs identify for new oral agents such as Catabasis Pharmaceuticals' edasalonexent and Italfarmaco's givinostat? And why are pipeline gene therapies such as Pfizer's PF-06939926, Sarepta Therapeutics/ Roche's SRP-9001, and Solid Biosciences' SGT-001 seen as game-changing? US and European KOLs critically assess the prospects of launched and pipeline therapies.

Therapy Trends reports give you an edge by delivering actionable intelligence to help you make informed decisions about your product. Based entirely on in-depth interviews with the world's foremost KOLs, Therapy Trends research focuses on the major battlegrounds for market share and answers critical business questions about product positioning and competitiveness.
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Key questions answered

  • Where do KOLs see value and position for casimersen, Sarepta's new exon 45 skipping agent?
  • What trial results could widen the use of Santhera Pharmaceuticals' Puldysa (oral idebenone), and in what settings?
  • To what extent do KOLs see PTC Therapeutics' price ambitions restricting the use of Translarna (ataluren) in limited nonsense mutation DMD population?
  • What advantages and challenges do KOLs identify for Nippon Shinyaku's Viltepso (viltolarsen)?
  • How has the COVID-19 pandemic impacted the DMD therapy sector?

Examples of Therapies Covered

Emflaza Puldysa
Edasalonexent Givinostat
Translarna Viltepso
Exondys 51 Vyondys 53
Casimersen PF-06939926
SGT-001 SRP-9001

Partial List of Participating KOLs

  • Professor of Neurology, Pediatrics, Obstetrics and Gynecology, Director of Pediatric Neuromuscular Medicine
    University of Rochester, Rochester, NY.
  • Associate Professor of Pediatrics and Associate Professor of Neurology at Duke Department of Pediatrics
    Duke University School of Medicine, Durham, NC.
  • Professor of Pediatrics, Vice Chair for Faculty Affairs, Department of Pediatrics
    Nationwide Children's Hospital, Columbus, OH.
  • Honorary Consultant in Human Genetics and Neurologist/Senior Research Associate at the Institute of Genetic Medicine,
    Newcastle upon Tyne Hospital, Newcastle, UK.
  • Professor/Senior Physician of Neuropediatrics in the Department of Women's and Children's Health (KBH)
    Division of Pediatric Neurology, Karolinska Institute, Stockholm, Sweden.
  • Professor of Neuromuscular Diseases, Head of Department of Neurology
    Leading University Medical Centre, The Netherlands.
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FirstWord PerspectivesOur-in house senior analysts and medics leverage their disease area knowledge and market research expertise to identify the key questions and issues.
FirstWord PerspectivesOur reports provide a detailed analysis, based on the candid views of world-renowned KOLs, of how treatment algorithms are likely to evolve over the next 3-5 years.
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