裘馨氏肌肉萎缩症(DMD)- KOL的见解

本报告对杜氏肌肉营养不良症 (DMD) 的现有和新兴治疗前景进行了全面分析，整合了美国和欧洲关键意见领袖 (KOL) 的观点以及最新的临床和商业数据。报告探讨了目前的标准疗法和后期研发管线疗法，包括类固醇、基因疗法、外显子跳跃药物、细胞疗法和新型小分子药物，重点介绍了它们的临床疗效、安全性和患者细分策略。报告也探讨了不断发展的治疗方案、支付者和可及性动态，以及联合疗法在满足未满足需求方面的潜力。

待解答的关键问题

• 医师目前如何治疗杜氏肌肉营养不良症 (DMD)？
• KOL 对目前的 DMD 治疗方案的评价是否令人满意？
• DMD 治疗如何因患者类型（例如年龄、疾病分期和遗传表型）而异？
• 目前 DMD 管理中尚未满足的需求
• 支付方和可及性课题影响 DMD 治疗决策
• 基因疗法和外显子跳跃疗法在多大程度上可能取代现有的 DMD 疗法？
• 新兴 DMD 疗法面临的长期课题，包括安全性、给药途径和成本。
• 下一代疗法是否会显着改善现有 DMD 疗法的疗效？
• 联合治疗策略可能如何塑造未来的 DMD 格局？

领导品牌

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.