MEDIPAL HOLDINGS CORPORATION (MEDIPAL) participated in the 21st Annual WORLDSymposium^TM 2025, held from February 3 to 7, 2025, at the Manchester Grand Hyatt in San Diego, California, United States.

“WORLDSymposium^TM” is an annual international conference held in the United States focusing on lysosomal storage diseases (LSDs). It serves as a platform for researchers, clinicians, patient advocacy groups, and other stakeholders involved with LSDs to share the latest research advancements (https://worldsymposia.org/).

This year’s symposium featured over 80 presentations, 10 corporate presentations, 350 poster presentations, and around 30 booths from companies and 10 patient advocacy groups related to lysosomal storage diseases. Attendees from around the world engaged in vibrant discussions regarding the latest research.

Since 2022, MEDIPAL has started new initiatives focusing on ultra-rare diseases, particularly those with very few patients among lysosomal storage diseases. This year marks our third consecutive participation in the symposium (2024 report).

Various companies, including pharmaceutical firms, showcased their booths at the symposium, while oral and poster presentations on the latest research were conducted. The presentations covered various topics, including new approaches to existing therapies, research on novel treatments without effective options for diseases, and efforts toward newborn screening in different countries. These presentations provided valuable insights. Notably, there was an increase in the number of presentations in the field of gene therapy, indicating advancements in this field.

Additionally, in collaboration with JCR Pharmaceuticals, we hosted several meetings with representatives from Cure Sanfilippo Foundation and ISMRD.

Our meeting with the Cure Sanfilippo Foundation facilitated a productive exchange of perspectives on global trends and approaches in drug development, and newborn screening in the United States. This discussion provided us with invaluable insights for future global development.

Our meeting with ISMRD representatives focused on topics related to the ISMRD 2025 meeting in August, and the current situation for patients with fucosidosis. We also discussed perspectives on future drug development strategies.

We are committed to deepening our understanding of rare diseases and collaborating with partner organizations to support patients and their families awaiting treatments.

The next WORLDSymposium^TM 2026 is set for February 1 to 5, 2026, in San Diego, California. We are scheduled to participate on-site.