デュシェンヌ型筋ジストロフィー（DMD）- KOLの見解

当レポートでは、デュシェンヌ型筋ジストロフィー（DMD）の現在の治療情勢と新たな治療情勢を包括的に分析し、米国と欧州のKOL（Key Opinion Leader）の見解を、最新の臨床データおよび商業データと統合しています。ステロイド、遺伝子治療、エクソンスキッピング薬剤、細胞治療、新規低分子など、現在の標準治療と後期パイプライン治療について検証し、その臨床効果、安全性プロファイル、患者セグメンテーション戦略を明らかにしています。また、進化する治療アルゴリズム、保険者とアクセスの力学、アンメットニーズに対応するための併用アプローチの可能性についても解説しています。

回答される主な質問

• 現在、医師はデュシェンヌ型筋ジストロフィー（DMD）をどのように治療しているか
• 現在のDMD治療オプションは、KOLから満足のいくものであると評価されているか
• 年齢、病期、遺伝的表現型などの患者タイプによって、DMD治療はどのように異なるのか
• 今日のDMD管理におけるアンメットニーズ
• DMD治療の意思決定に影響を与える保険者とアクセスの課題
• 遺伝子治療やエクソンスキッピング療法が、既存のDMD治療に取って代わる可能性はどの程度か
• 安全性、デリバリー、コストなど、新たなDMD治療の長期的課題
• 次世代の治療法は、現在のDMD治療に対して転帰を大きく向上させるか
• 併用治療戦略は、将来のDMD情勢をどのように形成する可能性があるか

主要ブランド

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.