裘馨氏肌肉萎縮症(DMD)- KOL的見解

本報告對杜氏肌肉營養不良症 (DMD) 的現有和新興治療前景進行了全面分析，整合了美國和歐洲關鍵意見領袖 (KOL) 的觀點以及最新的臨床和商業數據。報告探討了目前的標準療法和後期研發管線療法，包括類固醇、基因療法、外顯子跳躍藥物、細胞療法和新型小分子藥物，重點介紹了它們的臨床療效、安全性和患者細分策略。報告也探討了不斷發展的治療方案、支付者和可及性動態，以及聯合療法在滿足未滿足需求方面的潛力。

待解答的關鍵問題

• 醫師目前如何治療杜氏肌肉營養不良症 (DMD)？
• KOL 對目前的 DMD 治療方案的評價是否令人滿意？
• DMD 治療如何因患者類型（例如年齡、疾病分期和遺傳表型）而異？
• 目前 DMD 管理中尚未滿足的需求
• 支付方和可及性課題影響 DMD 治療決策
• 基因療法和外顯子跳躍療法在多大程度上可能取代現有的 DMD 療法？
• 新興 DMD 療法面臨的長期課題，包括安全性、給藥途徑和成本。
• 下一代療法是否會顯著改善現有 DMD 療法的療效？
• 聯合治療策略可能如何塑造未來的 DMD 格局？

領導品牌

This report provides a comprehensive analysis of the current and emerging therapeutic landscape for Duchenne muscular dystrophy (DMD), integrating insights from leading key opinion leaders in the US and Europe with the latest clinical and commercial data. It examines current standards of care and late-stage pipeline therapies-including steroids, gene therapies, exon-skipping agents, cell therapies, and novel small molecules-highlighting their clinical efficacy, safety profiles, and patient segmentation strategies. The report also explores evolving treatment algorithms, payer and access dynamics, and the potential of combination approaches to address unmet needs. Gain expert insights into the future direction of DMD therapy and the opportunities and challenges shaping the next generation of treatment.

Key Questions Answered:

• How do physicians currently treat Duchenne muscular dystrophy (DMD)?
• Are current DMD treatment options considered satisfactory by key opinion leaders?
• How does DMD treatment differ by patient type, such as age, disease stage, or genetic phenotype?
• What are the primary unmet needs in DMD management today?
• What payer and access challenges affect DMD treatment decisions?
• How likely are gene and exon-skipping therapies to displace established DMD treatments?
• What are the long-term challenges of emerging DMD therapies, including safety, delivery, and cost?
• Will next-generation therapies significantly improve outcomes over current DMD treatments?
• How might combination treatment strategies shape the future DMD landscape?

Key Brands:

Partial List of Participating Experts:

• Associate Professor of Neurology, Harvard Medical School and Associate, Department of Neurology at Boston Children's Hospital, Boston, MA
• Consultant in Paediatric Neurology and Neuromuscular Disorders at Evelina London Children's Hospital, St Thomas' Hospital, London, UK
• Pediatric neurologist and Assistant Professor of Neurology, Johns Hopkins Hospital/Kennedy Krieger Institute, Baltimore, MD
• Professor of Medical Genetics and Director of the Medical Genetics Unit at the Azienda Ospedaliero, University of Ferrara, Italy
• Professor of Medicine and Professor of Neuromuscular Genetics at the Institute of Translational and Clinical Research, Newcastle University, Newcastle, UK
• Professor of Pediatrics and Neurology at a leading university hospital and medical school in the USA

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.