Advanced Therapies Clinical Development


Advanced Therapies Clinical Development, Wednesday 24 April 2024

Adam Bloomfield, M.D.,  FAAP
Advanced Therapies Clinical Development

Unlocking Insights: How decentralized clinical trials and advanced therapies are revolutionizing pediatric rare disease research

  • This session will explore solutions to challenges in pediatric rare disease research, specifically in consideration of decentralized clinical trials (DCT) and cell and gene therapy development. You will learn:
  • The complexities of executing cell and gene therapy programs in pediatric populations
  • Key considerations for instituting a DCT approach for cell and gene therapy programs in a pediatric population
  • Strategies for seamless operationalization of DCTs
Renata Gallagher, MD, PhD
Advanced Therapies Clinical Development

An honest discussion: The challenges of genotype interpretation and phenotype variation in rare disease

Linda Dell
Advanced Therapies Clinical Development

From science to solution: Navigating the clinical development journey

Advanced Therapies Clinical Development

Maximizing therapeutic impact: Exploring the potential of basket trials for concurrent treatment strategies

Panel discussion
Laurence Mignon
Advanced Therapies Clinical Development

Treating the nano-rare: Redefining clinical management, one patient at a time

Panel discussion
Advanced Therapies Clinical Development

Conducting clinical studies in rare diseases

Moderator: Marshall Summar, Chief Executive Officer, Uncommon Cures
Ron Bartek, President, Co-Founder, Friedreich's Ataxia Research Alliance
last published: 20/Apr/24 16:35

Advanced Therapies Clinical Development, Thursday 25 April 2024

last published: 20/Apr/24 16:35




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