8th Annual Symposium

GRIDS 2022

Downstream Pathways and Lysosomal Storage Disorders: From Translational to Clinical Contexts

November 20–21, 2022 · Fairfax, Virginia, USA

Global Learning Objectives

  1. Learn the molecular and cellular mechanisms behind lysosomal function, and how they relate to the pathology and progression of lysosomal storage disorders (LSDs).
  2. Understand the burden and role of inflammatory pathways that lead to cell death and the manifestation of LSDs.
  3. Learn about the role of genomic and metabolomic approaches in precision, or individualized, medicine.
  4. Gain insight into current treatment strategies, findings from preclinical studies in different model systems, and clinical trials of novel therapies.
  5. Discuss the efficacy and safety of clinical studies of next-generation enzyme replacement therapies that use hyper-mannosylation and other techniques, such as antibody peptides and targeting sequences, to improve tissue penetration.
  6. Review pharmacological chaperones and small-molecule approaches as disease-specific or symptomatic treatments for various LSDs.
  7. Learn about novel tools and technologies that incorporate big data and machine learning.
  8. Discuss the clinical utility of sphingolipid and other biomarkers in the diagnosis and therapeutic management of LSDs.

Enduring CMEs

Each enduring CME session offers up to 2 hours of CME credit.

  • Session 1

    Achievements and Disappointments: Gaucher Disease Over the Past 3 Decades

    Three decades of Gaucher disease research and treatment: therapeutic breakthroughs, ongoing challenges, and lessons learned.

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  • Session 2

    Basic Mechanisms and Model Systems in Lysosomal Disorders

    The molecular and cellular mechanisms underlying lysosomal dysfunction, with insights from a range of preclinical model systems.

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  • Session 3

    Application of Novel Technologies in Lysosomal Disorders

    How big data, machine learning, and advanced platforms are reshaping research and clinical practice in lysosomal storage disorders.

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  • Session 4

    Biomarkers and Other Genetic/Genomic Approaches in Lysosomal Disorders

    Sphingolipid biomarkers, genomics, and metabolomics for precision medicine and improved therapeutic management of LSDs.

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  • Session 5

    Novel and New-Generation Therapies in Lysosomal Disorders

    Next-generation enzyme replacement therapies, pharmacological chaperones, and small-molecule therapies designed for better tissue penetration and disease-specific treatment.

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  • Session 6

    Current Clinical Management and Challenges in Lysosomal Disorders

    Real-world clinical perspectives on diagnostic delays, treatment access, monitoring strategies, and persistent unmet needs in LSD care.

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