Description
+ Include: 1 videos + 7 pdfs, size: 767 MB
+ Target Audience: physicians, geneticists, neurologists, pediatricians
+ Sample video: contact me for sample video
+ Information:
This continuing medical education conference provides a comprehensive update on lysosomal storage disorders (LSDs) for multidisciplinary healthcare teams. LSDs are genetic conditions caused by deficiencies in lysosomal components, primarily enzymes, which lead to lysosomal dysfunction and have widespread effects on peripheral organs and the central nervous system. Participants will explore the latest advances in the identification and diagnosis of LSDs, as well as current understanding of their pathophysiology. The course will also cover updates on management strategies and treatment options, emphasizing the translation of scientific breakthroughs into clinical practice. Expert faculty will present various clinical cases for discussion, along with research updates and case reports that highlight recent developments in the field. This conference aims to enhance the ability of healthcare professionals to provide optimal care for patients with these rare but debilitating disorders, equipping attendees with up-to-date knowledge to improve early diagnosis, implement appropriate management plans, and incorporate emerging therapies into their clinical practice.
- Apply updated knowledge of lysosomal storage disorders to improve diagnostic accuracy, develop prognoses, contribute to research, and enhance patient care
- Identify the association between Gaucher disease and cancer
- Describe the benefits, risks and limitations of newborn screening for lysosomal storage disorders in California
- Analyze and integrate knowledge of Lysosomal Storage Disorders (LSDs) to collaborate effectively within interprofessional teams, facilitating improved patient care outcomes.
The Cedars-Sinai 15th Annual Lysosomal Storage Disease Symposium 2025 is best for physicians, geneticists, neurologists, pediatricians, and multidisciplinary healthcare providers who manage patients with lysosomal storage disorders (LSDs). It provides updates on diagnosis, treatment, and research advances, making it especially valuable for clinicians working in metabolic medicine, neurology, pediatrics, and rare disease care.
👩⚕️ Who Should Attend
- Physicians & specialists in neurology, pediatrics, internal medicine, and genetics.
- Metabolic disease experts managing LSDs such as Gaucher, Fabry, Pompe, and MPS disorders.
- Advanced practice providers (NPs, PAs) working in rare disease or neurology clinics.
- Researchers & academic faculty studying lysosomal biology and therapeutic innovations.
- Multidisciplinary teams (nurses, pharmacists, therapists) involved in patient care.
📚 What You’ll Learn
- Latest advances in LSDs: updates on pathophysiology, genetics, and clinical manifestations.
- Therapeutic innovations: enzyme replacement therapy, substrate reduction, gene therapy.
- Multisystem involvement: neurological, cardiac, skeletal, and visceral complications.
- Case‑based learning: practical approaches to diagnosis and long‑term management.
- Future directions: clinical trials, biomarkers, and personalized medicine strategies.
+ Topics:
- Recording.mp4
- Welcome and Introduction.pdf
- Breakthroughs in Treatment for LSDs.pdf
- Cancer in Gaucher Disease.pdf
- Clinical Case 1.pdf
- Clinical Case 2.pdf
- Gaucher Disease – Clinical Updates.pdf
- Gaucher Disease – Research Updates.pdf
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