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Conference Program

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Rare Disease Therapeutics: From Bench to Bedside

14 Mar 2024
Venue: Metro Toronto Convention Center
Meeting Room: 801 AB
Clinical Genetics and Therapeutics
  • Accredited:
    • Accredited
  • Primary Categories:
    • Research
  • Secondary Categories:
    • Research
  • Level of Learner:
    • Intermediate
With the rapid increase in rare disease therapeutics, medical genetics providers are frequently faced with questions about the therapeutic potential for investigational therapies, & the potential risks/benefits of newly-approved therapies. It has therefore become important to be familiar with the drug development life cycle. This session will include key topics that are relevant to basic science and clinical investigators & genetics providers. The topics are organized around three facets of drug development.
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Learning Objectives

  1. Explain the impact of preclinical studies in assessing the potential for successful translation to humans
  2. Review the data needed to support development of drugs for rare diseases
  3. Identify specific areas of clinical study design that benefit from participant input
  4. Recognize important ethical considerations for the protection of vulnerable populations in clinical research
  5. Review relevant considerations in development of efficacy endpoints in ultra-rare conditions
  6. Justify the prospect of direct benefit from development of drugs for rare diseases