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Conference Program

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Lysosomal Storage Diseases - Emerging Diagnostics and Therapies

16 Mar 2024
Venue: Metro Toronto Convention Center
Meeting Room: 801 AB
Biochemical/Metabolic and Therapeutics
  • Accredited:
    • Accredited
  • Primary Categories:
    • Metabolic Genetics
  • Secondary Categories:
    • Metabolic Genetics
  • Level of Learner:
    • Intermediate
This session will first examine considerations related to newborn screening for LSDs. We will review updates regarding fetal diagnosis including sonographic findings, challenges related to fetal sequencing, and the impact of carrier screening. An overview of an ongoing phase 1 clinical trial of in utero-ERT for LSD patients will be provided, as well as an overivew of other potential prenatal LSD therapies. We will then outline current and emerging therapies available postnatally (including gene therapies), with a focused overview of the strategies and use of HSCT. Finally, we will provide a discussion regarding advocacy and patient engagement to reduce the barriers for optimal LSD therapies.
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Learning Objectives

  1. Dene LSDs screened for by newborn screening and the opportunities and challenges of expanding to more conditions
  2. Recognize prenatal presentations of LSDs, carrier screening, and prenatal diagnosis options
  3. Describe the current and emerging treatment strategies pre and postnatally to optimize outcomes in LSDs
  4. Detail the status of hematopoietic stem cell transplantation for LSDs including outcomes and alternatives
  5. Define the regulatory and access barriers for novel therapies in LSDs

Agenda

  • N. Matthew Ellinwood, DVM, PhD
    Regulatory and Access Barriers for Novel Therapies in Lysosomal Storage Diseases
    8:00 AM – 8:15 AM
  • Muhammad Ali, MD
    The Role of Hematopoietic Stem Cell Transplantation for LSDs
    8:15 AM – 8:30 AM
  • Billie Lianoglou, MS
    Prenatal Diagnosis and Emerging Fetal Therapies for Lysosomal Storage Diseases
    8:30 AM – 8:45 AM
  • Pranesh Chakraborty, MD, FRCPC, FCCMG
    Newborn Screening Considerations for Lysosomal Storage Diseases
    8:45 AM – 9:00 AM
  • Paul R. Harmatz, MD
    Current and Emerging Postnatal Therapies for Lysosomal Storage Diseases
    9:00 AM – 9:15 AM
  • Panel Discussion
    Panel Discussion and Q&A
    9:15 AM – 9:30 AM

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