What’s New in NF1: Recent Research Advances, Emerging Therapies, and Future Perspectives

In this presentation, Dr. Julien Rousseau, neuro-oncology fellow in Boston and soon to be joining the CHUM, presents the latest research advances and emerging therapeutic approaches in NF1. He provides an overview of new treatments, ongoing studies, and the evolving prospects for the future. This presentation sheds light on the rapid progression of the field and the hope it brings for patients and families.
Julien rousseau

Conference – What’s New in NF1: Recent Research Advances, Emerging Therapies, and Future Perspectives (French only)

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Here is an overview of the topics covered in this presentation:

  • Historical evolution of NF1 treatments
  • MEK inhibitors
    • Selumetinib: the first major breakthrough
    • Mirdametinib: new approval in 2025
  • Topical treatments for NF1
  • Photographic measurement of neurofibromas
  • Immunotherapy in NF
  • Gene therapy
  • Advanced imaging in NF1
  • Cognitive challenges in NF1
  • Multidisciplinary care
  • The future of NF1
    • Ongoing studies
    • Platform trials: a methodological revolution
  • Key take-home messages
recherche et traitement