GRIDS 2022
Downstream Pathways and Lysosomal Storage Disorders:
From Translational to Clinical Contexts
November 20th – 21st, 2022
Fairfax, Virginia, USA
Educational Goals
Global Learning Objectives
- Get introduced to molecular and cellular mechanisms as related to different lysosomal functions and disease pathology and progression in LSDs.
- Gain knowledge about the burden/role of inflammatory pathways leading to cell death and the manifestation of LSDs.
- Get introduced to the role of genomics and metabolomics approaches in precision or individualized medicine.
- Gain insights into current treatment strategies and findings from preclinical studies in different model systems and clinical trials towards novel therapies.
- Discuss the efficacy and safety of clinical studies involving next-generation enzyme replacement therapies that utilize hyper-mannosylation and other techniques such as antibody peptides and targeting sequences for improved tissue penetration.
- Review pharmacochaperones and small molecular approaches as disease-specific or symptomatic treatment of various LSDs.
- Learn about novel tools and technologies that incorporate big data and machine learning.
- Discuss the clinical utility of sphingolipid and other biomarkers in the diagnosis and therapeutic management of LSDs.
Continuing Education
Enduring CMEs
Enduring CME with up to 2 hours of CME credit per session.
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.
Access CME →Basic Mechanisms and Model Systems in Lysosomal Disorders
Molecular and cellular mechanisms underlying lysosomal dysfunction, with insights from various preclinical model systems.
Access CME →Application of Novel Technologies in Lysosomal Disorders
Big data, machine learning, and advanced platforms reshaping research and clinical practice in lysosomal storage disorders.
Access CME →Biomarkers and Other Genetic/Genomic Approaches in Lysosomal Disorders
Sphingolipid biomarkers, genomics, and metabolomics for precision medicine and improved therapeutic management of LSDs.
Access CME →Novel and New Generation Therapies in Lysosomal Disorders
Next-generation ERTs, pharmacochaperones, and small molecule therapies engineered for improved tissue penetration and disease-specific treatment.
Access CME →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.
Access CME →