The inaugural Robert J. Gorlin Symposium discussed the urgent need to develop and implement precise metrics for measuring cognitive change in children with lysosomal diseases. Precision Metrics for Cognition brought together leading experts to provide an explanation and rationale for using growth scale values (GSVs) to measure developmental longitudinal cognitive change in children with early onset and severe lysosomal diseases. More details.
The goal of the 2023 Symposium was to address the tremendous opportunity for Precision Medicine in lysosomal diseases. Over the past decade there has been much discussion about “Precision Medicine” and its use for many different diseases, including lysosomal disorders. Although it is not a new concept, more and more researchers and clinicians are recognizing that precision medicine must guide the path forward for patients with lysosomal diseases. More details.
The 2024 session discussed current approaches for treating the central nervous system in lysosomal diseases. Presentations included an overview of current and future approaches for treating the central nervous system, and address challenges in identifying and implementing meaningful outcome measures for evaluating efficacy of central nervous system therapies. More details.