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 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, 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.

GRIDS2022 Enduring

Achievements and Disappointments: Gaucher Disease Over the Past 3 Decades

https://checkrare.com/learning/p-grids2022-session1-achievements-and-disappointments-gaucher-disease-over-the-past-3-decades

Application of Novel Technologies in Lysosomal Disorders

https://checkrare.com/learning/p-grids2022-session3-application-of-novel-technologies-in-lysosomal-disorders/

Biomarkers and Other Genetic/Genomic Approaches in Lysosomal Disorders

https://checkrare.com/learning/p-grids2022-session4-biomarkers-and-other-genetic-genomic-approaches-in-lysosomal-disorders/

Novel and New Generation Therapies in Lysosomal Disorders

https://checkrare.com/learning/p-grids2022-session5-novel-and-new-generation-therapies-in-lysosomal-disorders/

Current Clinical Management and Challenges in Lysosomal Disorders

https://checkrare.com/learning/p-grids2022-session6-current-clinical-management-and-challenges-in-lysosomal-disorders/

Basic Mechanisms and Model Systems in Lysosomal Disorders

https://checkrare.com/learning/p-grids2022-session2-basic-mechanisms-and-model-systems-in-lysosomal-disorders/