KOL Insight: Spinal Muscular Atrophy [2020]

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Publication Date:
October 2020
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Multi-user licence
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Therapy Trends KOL Insight: Spinal Muscular Atrophy [2020]

Despite its price, KOLs see Novartis' Zolgensma as a 'game changer' in infant SMA but will safety concerns around the intrathecal formulation slow its expansion in older populations? And as Roche/PTC Therapeutics' Evrysdi impacts the market, what role do KOLs see for Biogen/Ionis Pharmaceuticals' established market leader Spinraza? 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

  • Do experts see a future for Novartis' pipeline SMA oral therapy branaplam?
  • Why do KOLs see the results of SPR1NT, STR1VE and START studies for Zolgensma as impressive?
  • What's the case for continuing treatment with Spinraza after Zolgensma and will payers be prepared to meet the bill?
  • How do KOLs assess the prospects for add-on pipeline SMA therapies such as Scholar Rock's SRK-015, Astellas/Cytokinetics' reldesemtiv and BioMarin/Catalyst Pharmaceuticals' amifampridine?

Examples of Therapies Covered

Spinraza Evrysdi
Zolgensma Reldesemtiv
Branaplam Amifampridine

Partial List of Participating KOLs

  • Professor of Neurology; Associate Neurologist-in-Chief; Chief of Clinical Neurology; Director
    Neuromuscular Center and Spinal Muscular Atrophy Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, US
  • Professor of Neurology; Medical Director
    Center for Noninvasive Mech Ventilation, Rutgers New Jersey Medical School, Newark, New Jersey, US
  • Professor of Neurology, Pediatrics and Obstetrics and Gynecology; Director of Pediatric Neuromuscular Medicine
    University of Rochester, New York, US
  • Professor of Paediatric Neuromuscular Diseases
    MDUK Oxford Neuromuscular Centre, University of Oxford, UK
  • Paediatric Neurologist; Head of the Unit of Neuromuscular and Neurodegenerative Disorders
    Bambino Gesu' Children's Research Hospital, Rome, Italy
  • Professor in Neuropediatrics, Consultant Paediatrician
    Department of Women's and Children's Health of Karolinska Institute, Astrid Lindgrens Barnsjukhus, Solna, Sweden
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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.
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