Technology and Rare Neurological Diseases Symposium

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Presented By: University of Rochester
Friday, September 13, 2019
7:30 am – 4:00 pm

Location: University of Rochester Saunders Research Building | 265 Crittenden Boulevard | Rochester, NY 14620

The University of Rochester, a national leader in rare diseases clinical research and therapeutic development, is hosting the second bi-annual TRNDS meeting. TRNDS is a one-day symposium on emerging novel therapeutic technologies that are creating new and rapidly expanding treatment options for patients with rare neurological disorders.

The symposium will assemble regional and national leaders in rare diseases and therapy development to discuss key concepts and controversies surrounding new neurological therapies including: exploration of ethical considerations in testing of novel therapies, challenges of long-term follow-up, and avenues to early diagnosis.

Speakers include:

  • Leading clinical researchers
  • Forward-thinking health technology leaders
  • Influential patient advocates
  • Experts in regulatory science

Topics include:

  • Emerging gene-based therapies for neurological disorders
  • The pace of newborn screening expansion and keeping up with therapeutic development  The cost of technological advancement  Efficiencies in the application of novel technologies for the advancement of clinical research
  • New tools for natural history studies, including the electronic health record
  • Wearable sensors, smartphone applications and new patient-reported outcomes
  • Site-less clinical trials and virtual research visits
  • Ethical considerations in the modernization of clinical trials

Participants will:

  • Learn the latest research regarding health technologies in rare diseases
  • Discover new ideas and application of technology to clinical trials and clinical research
  • Join a community of experts dedicated to accelerating the discovery of new therapies for patients with rare neurological disorders

Draft Content:
Panel 1: Gene Therapy – what have we learned? What is the future potential for rare neurological diseases?
Panel 2: Are treatment advances outpacing advances in early and efficient diagnosis?
Panel 3: Investigational therapies ready for testing without sufficient disease knowledge - Modern approaches to natural history studies
Panel 4: The cost of success
Keynote: Generating the path to therapeutic victories for rare diseases

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