9th Annual Symposium

GRIDS 2023

From Asymptomatic to Severe, the Variability in Lysosomal Storage Disorders: Integrating the Basics with Current Clinical Practices

November 19–20, 2023 · Salamander Middleburg (opens in a new tab), Middleburg, Virginia, USA

9th Annual GRIDS Symposium

Lysosomal disorders pose significant challenges for therapy because of their complex nature and phenotypic variability. Advances in understanding lysosomal biology have shed light on the lysosome’s dynamic role in cellular function and in the development of pathology. With the emergence of novel technologies such as gene therapy and genome editing, along with advances in diagnostics, biomarkers, and companion diagnostics, the field is evolving rapidly. The Genetic, Rare and Immune Disorders Symposium (GRIDS) 2023 brings together clinicians, researchers, and advocacy groups to exchange knowledge, promote collaboration, and improve the diagnosis and treatment of lysosomal disorders, ultimately improving the lives of patients.

Program highlights

  • Basic Mechanisms in Lysosomal Disorders: Phenotypic Diversity and Cell Biology
  • Biomarkers and Other Genetic Approaches in Lysosomal Storage Disorders (LSDs)
  • The Application of Novel Technologies in LSDs: The Role of AI in Lysosomal Disorders
  • Next-Generation Therapies in LSDs
  • Current Clinical Challenges in Lysosomal Disorders: Phenotypic Diversity and Clinical Management
  • Newborn Screening and LSDs in the Neonatal Period

Speakers

Keynote speaker

Konrad SandhoffPhD

University of Bonn, Germany

Confirmed speakers

  • Aimee DonaldPhD

    NIHR Academic Clinical Lecturer, University of Manchester, UK

  • Alexey V. PshezhetskyPhD

    Departments of Pediatrics and Biochemistry, University of Montreal, Canada

  • Andrea DardisPhD

    University Hospital Santa Maria della Misericordia, Italy

  • Can FiciciogluMD, PhD

    Children’s Hospital of Philadelphia (CHOP), Pennsylvania, USA

  • Caroline HastingsMD

    Children’s Hospital & Research Center Oakland, California, USA

  • David KronnMD

    Maria Fareri Children’s Hospital, New York Medical College, USA

  • Debra S. RegierMD, PhD

    Children’s National Medical Center, Washington, DC, USA

  • Elizabeth JalazoMD

    University of North Carolina, USA

  • Gregory A. GrabowskiMD

    University of Cincinnati College of Medicine, Ohio, USA

  • Guilherme BaldoPhD

    Universidade Federal do Rio Grande do Sul, Brazil

  • Gustavo MaegawaMD, PhD

    Columbia University Irving Medical Center, New York, USA

  • John Lynn JefferiesMD

    University of Tennessee Health Science Center, USA

  • Joseph MuenzerMD, PhD

    University of North Carolina, USA

  • Magy Abdel WahabMD

    Cairo University Pediatric Hospital, Egypt

  • Mia HorowitzPhD

    Tel Aviv University, Israel

  • Orna Staretz-ChachamMD

    Soroka Medical Center, Israel

  • Pilar GiraldoMD

    Instituto de Investigación Sanitaria Aragón, Spain

  • Shawn M. FergusonPhD

    Yale School of Medicine, Connecticut, USA

  • Shoshana Revel-VilkMD

    Shaare Zedek Medical Center, Jerusalem, Israel

  • Neil Kasaci

    LDRTC, Virginia, USA

    Young Investigator

Agenda Overview

Day 1

November 19, 2023
  1. IBasic Mechanisms in Lysosomal Disorders: Phenotypic Diversity and Cell Biology
  2. IIBiomarkers and Other Genetic Approaches in LSDs
  3. IIICurrent Clinical Challenges in Lysosomal Disorders: Newborn Screening and LSDs in the Neonatal Period
  4. IVThe Application of Novel Technologies in LSDs: The Role of AI in Lysosomal Disorders

Day 2

November 20, 2023
  1. VNext-Generation Therapies in LSDs
  2. VICurrent Challenges in Lysosomal Disorders

Enduring CMEs

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