MEDIPAL HOLDINGS (MEDIPAL) participated in the 7th International Glycoproteinoses Family and Scientific Forum, held at Graduate Minneapolis in Minneapolis, Minnesota, USA, from August 8 to 9, 2025.

The forum, organized by ISMRD, a patient advocacy group for Glycoprotein Storage Diseases*, focuses on lysosomal storage diseases with particularly low patient numbers. It aims to provide global support through partnerships and information on diagnosis and treatment in medicine, science, and industry.

*Glycoprotein Storage Diseases (glycoprotein metabolism disorders) are a group of lysosomal storage diseases caused by defective degradation of glycoproteins in lysosomes. These diseases include the following: fucosidosis, α-mannosidosis, β-mannosidosis, aspartylglucosaminuria, galactosialidosis, mucolipidosis type II, mucolipidosis type III, sialidosis, and Schindler disease/Kanzaki disease. Other than α-mannosidosis, there are currently no approved treatments for these conditions.

This year, approximately 180 participants, including patients, their families, physicians, and pharmaceutical companies from around the world, attended either in-person or virtually. Discussions transcended the boundaries of various fields and covered topics including disease characteristics, biomarkers linked to treatment effectiveness, and challenges and potential solutions of drug development.

It was our first time to participate in the International Glycoproteinoses Family and Scientific Forum, following the start of a new initiative in the field of ultra-rare diseases with JCR Pharmaceuticals Co., Ltd., in 2022.

During the meeting, information on the pathology and updates on the current treatment status for each disease were shared. Also, there was a lively exchange of opinions on measurable endpoints for verifying treatment effectiveness in glycoprotein storage diseases, the usefulness of the Scale for Assessment and Rating of Ataxia (SARA), the importance of early diagnosis and early treatment, and the challenges of implementing newborn screening. Discussions throughout the forum focused on how patients, healthcare professionals, and companies could collaborate, fostering a sense of unity in the shared mission to confront these diseases for which there are no treatments.

In this collaborative atmosphere, Carolyn, the former representative of the patient advocacy group, expressed her deep gratitude to MEDIPAL/JCR for developing a treatment.

We are committed to deepening our understanding of rare diseases and collaborating with partner organizations to contribute to patients and their families who are waiting for treatments.