한글목차

이 보고서는 미국과 유럽의 주요 오피니언 리더들의 인사이트와 최신 임상 및 상업 데이터를 통합하여 듀셴형 근이영양증(DMD)의 현재 및 새로운 치료 환경에 대한 종합적인 분석을 제공합니다. 이 보고서는 스테로이드, 유전자 치료제, 엑손 스킵 제제, 세포 치료제, 새로운 소분자 등 현재 표준 치료법과 후기 단계 파이프라인 치료법을 조사하여 임상 효능, 안전성 프로필, 환자 세분화 전략을 강조합니다. 이 보고서는 또한 진화하는 치료 알고리즘, 지불자 및 접근성 역학, 충족되지 않은 요구를 해결하기 위한 조합 접근법의 잠재력을 탐구합니다. 다발성 경화증 치료의 미래 방향과 차세대 치료의 기회와 과제에 대한 전문가의 인사이트를 얻으세요.

답변을 얻을 수 있는 주요 질문

• 현재 의사들은 듀센 근이영양증(DMD)을 어떻게 치료하고 있는가?
• 주요 오피니언 리더들은 현재의 DMD 치료 옵션이 만족스럽다고 생각하는가?
• 연령, 질병 단계 또는 유전적 표현형 등 환자 유형에 따라 DMD 치료는 어떻게 다른가?
• 오늘날 DMD 관리에서 충족되지 않은 주요 요구사항은 무엇인가?
• DMD 치료 결정에 영향을 미치는 지불자 및 접근성 문제는 무엇인가?
• 유전자 및 엑손 건너뛰기 치료법이 기존 DMD 치료법을 대체할 가능성은 얼마나 될까?
• 안전성, 전달, 비용 등 새로운 DMD 치료법의 장기적인 과제는 무엇인가?
• 차세대 치료법이 현재의 DMD 치료법보다 결과를 크게 개선할 수 있는가?
• 병용 치료 전략은 미래의 DMD 환경을 어떻게 변화시킬 수 있는가?

주요 브랜드

영문목차

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.