MEDIPAL HOLDINGS CORPORATION (MEDIPAL) attended the 22nd Annual WORLDSymposium^TM 2026, held from February 2 to 6, 2026 at the Manchester Grand Hyatt in San Diego, California, United States.

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

This year’s symposium featured over 80 presentations, 10 satellite symposia, and more than 400 poster presentations. With exhibits from approximately 30 industry partners and 10 patient advocacy groups, attendees from around the world engaged in dynamic discussions on the latest research in the field.

Since 2022, MEDIPAL has started new initiatives focused on ultra-rare diseases, specifically those with extremely small patient populations among lysosomal storage diseases. This year marks our fourth consecutive year of participation in the symposium (2025 report).

Throughout the event, we attended a wide range of platform presentations and poster sessions covering the latest research and ongoing clinical trials in the field of LSDs.
The scope of these sessions was remarkably extensive, ranging from advancements in increasing infusion rates for existing therapies to the cutting-edge gene therapy development for conditions that currently have no approved treatment options. Furthermore, we gained insights into Newborn Screening (NBS) designed to make early detection both simpler and more efficient. These sessions provided us with a deeper understanding of LSDs.

In collaboration with JCR Pharmaceuticals, we also engaged with several prominent patient advocacy groups, including the Cure Sanfilippo Foundation, the International Society for Mannosidosis & Related Diseases (ISMRD), the National Tay-Sachs & Allied Diseases Association (NTSAD), the National MPS Society, the International MPS Network (IMPSN), and the International Sanfilippo Syndrome Alliance (ISSA).

These discussions highlighted the significant global disparities in NBS, noting that both the specific diseases screened and implementation rates vary widely by region. We also gained valuable insights into the advocacy groups’ perspectives on expanding screening programs and their proactive efforts to accelerate the delivery of novel treatments. These conversations reaffirmed that drug development requires more than just corporate effort; it is built upon a vital partnership between companies, clinicians, and the patient community.

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 2027 is set for January 31 to February 4, 2027, in San Diego, California. We are scheduled to participate on-site.