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PUBLISHER: FirstWord Group | PRODUCT CODE: 1815991

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PUBLISHER: FirstWord Group | PRODUCT CODE: 1815991

KOL Insight - Spinal Muscular Atrophy

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This report provides a comprehensive analysis of the current and future treatment landscape for spinal muscular atrophy (SMA), focusing on biologic and pipeline therapies. It presents insights from interviews with key opinion leaders (KOLs) on the market dynamics of Zolgensma, Evrysdi, and Spinraza, and explores emerging therapies like salanersen and anti-myostatin agents. Gain an understanding of the impact of newborn screening on therapy uptake, the competitive positioning of oral and gene therapies, and the potential for novel add-on treatments. The report also delves into the evolving treatment pathways and the implications of new clinical data, providing pharmaceutical professionals with a detailed view of the SMA therapeutic environment.

Key Questions Answered:

  • 1. How extensively has Zolgensma penetrated the SMA market and what patient populations is it attracting?
  • 2. What impact is the expansion of newborn screening having on the uptake of Zolgensma?
  • 3. How successfully is Evrysdi competing with Spinraza and Zolgensma in the SMA treatment landscape?
  • 4. What are the current usage trends and challenges associated with Spinraza?
  • 5. How are the currently marketed therapies Zolgensma, Evrysdi, and Spinraza expected to evolve over the next three to five years?
  • 6. What advantages does salanersen offer compared to Spinraza, and can it compete effectively with current therapies?
  • 7. How excited are KOLs about the potential of anti-myostatin therapies in treating SMA?
  • 8. What is the potential role of NMD Pharma's bromophenoxyazole propanoic acid in the SMA treatment paradigm?
  • 9. Are combination drug regimens expected to be adopted for SMA, and how will they advance patient outcomes?

Key Brands:

  • Novartis
  • Biogen
  • NMD Pharma
  • Biohaven
  • Roche
  • Scholar Rock
  • Chugai
  • IONIS pharmaceuticals

Methodology:

Therapy Trend reports are developed through in-depth interviews with the world's foremost Key Opinion Leaders (KOLs) on the current and future treatment landscapes in major disease areas. KOLs are carefully selected based on strict screening criteria, including their global reputation, clinical expertise, and influence within their therapeutic area. Each interview is guided by a meticulously crafted discussion guide. These guides are developed in collaboration with KOLs and peer-reviewed by industry experts to ensure that the questions are comprehensive and pertinent to current market dynamics. Through continuous market monitoring over a 12-month period following the publication of each report, we provide timely updates from KOLs on key news events, market shifts, and clinical developments.

What makes our reports different?

FirstWord Reports, a trusted industry leader with an exclusive focus on the pharmaceutical sector, delivers in-depth, actionable insights for biopharma professionals and decision-makers. Our deep industry knowledge enables us to provide relevant and valuable insights that help you identify emerging trends and navigate complex challenges effectively. Backed by extensive research and independent, impartial insights from leading experts and KOLs, our reports deliver the accuracy and reliability you need. With exclusive access to interviews and data unavailable elsewhere, along with ongoing market monitoring, we give you a comprehensive view of market dynamics. Covering over 40 dynamic disease areas and providing physician intelligence, including KOL insights and quantitative physician surveys, as well as industry expert views on issues in medical affairs, digital health, sales & marketing, market access, and other areas, our reports empower you to make smarter, data-driven decisions and maintain a competitive edge in a fast-moving industry.

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Jeroen Van Heghe

Manager - EMEA

+32-2-535-7543

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Christine Sirois

Manager - Americas

+1-860-674-8796

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