PICK UPTopics on rare diseases

We have selected information about rare diseases that we desire for everyone to know.

GM2 Gangliosidoses
2026.02.02
- #Lysosomal storage diseases
- #GM2 Gangliosidoses
- #Tay-Sachs disease
- #Sandhoff disease
Basic Knowledge of Lysosomal Storage Diseases－Overview
2025.02.03
- #Lysosomal storage diseases
- #Overview

REPORT & INFORMATIONActivities Report / Information

We provide a wide range of useful information on rare diseases, especially in the area of lysosomal storage diseases.

Research and Development of Lysosomal Storage Diseases ^NEW
2026.10.01
- #Lysosomal storage diseases
- #Clinical trial for regulatory submission
- #Clinical trial
International Mucopolysaccharidosis (MPS) Awareness Event 2026 Held for Employees
2026.08.03
- #Rare disease
- #Mucopolysaccharidosis
- #International MPS Awareness Day
- #Disease awareness
"Rare Disease Week 2026" disease awareness event held for employees
2026.05.07
- #Rare disease
- #Rare disease day
- #Disease awareness
22nd Annual WORLDSymposium^TM 2026 Report
2026.02.27
- #Lysosomal storage diseases
- #Participation report
- #Patient association
- #Mucopolysaccharidosis type III
- #Fucosidosis
- #GM2 Gangliosidoses
GM2 Gangliosidoses
2026.02.02
- #Lysosomal storage diseases
- #GM2 Gangliosidoses
- #Tay-Sachs disease
- #Sandhoff disease
Terms
2025.12.01
- #Lysosomal storage diseases

Article list

WHAT WE WANT TO DELIVEROur target initiatives

Provide new medications throughout the world

It is our desire to provide new medications to patients around the world.

Deliver medications and information

We desire to provide medications and information to each and every person who needs it.

ABOUTOur desires

Make you smile, Brighten your future

We put up “Expand, Support, and Connect ‘Pharmaceuticals, Health, and Beauty’” as the “ideal situation”, and we are developing various business activities to contribute to the healthy lives of people. For rare diseases, we have been making use of our Group’s strong distribution capabilities by providing development support to pharmaceutical companies ("PFM^Ⓡ") and establishing ultra-low temperature control logistics technology. Since 2022, as a new initiative, we have been taking on the challenge of developing pharmaceuticals in cooperation with JCR Pharmaceuticals Co., Ltd. and delivering to patients globally in the field of ultra-rare diseases, where the number of patients is particularly small.

The theme of this website is “Make you smile, Brighten your future,” and we will provide useful information to bring smiles to as many patients with rare diseases and their families as possible. By exchanging and disclosing information with patients, their families, and healthcare professionals, we hope to develop this site as a place where patients, medical treatment, pharmaceuticals, and distribution can unite and connect.

PFM^Ⓡ：Project Finance & Marketing
A business model where risks and returns are shared with pharmaceutical companies through investment in medication development

Image of medical care that empathizes with patients

The disease is known to exist, and the doctors exist.
But there is no treating medication.
What can we do to address the realities of rare diseases?
We believe that this means being close to the desires of the patients and their families, and developing and reliably delivering innovative pharmaceuticals that are not yet available in the world. The MEDIPAL Group will continue to take on the challenge not only in Japan but also on a global scale of nurturing invaluable life by envisioning the healthy upbringing of the world's children who will carry our future.

MEDIPAL HOLDINGS CORPORATION Representative Director, Chairman Shuichi Watanabe — Shuichi Watanabe
Representative Director,
Chairman
MEDIPAL HOLDINGS CORPORATION

“Bring us hope through new treatments—without a moment to lose.” This is the urgent wish of patients and their families.
In ultra-rare disease areas such as lysosomal storage diseases (LSDs), critical medical needs remain unmet.
Confronting this reality head-on, the MEDIPAL Group is dedicated to building a supportive healthcare ecosystem. We connect healthcare professionals and partners across every step of the journey—from supporting the development of therapies yet to be discovered and ensuring their reliable delivery, to enabling early diagnosis and providing patient care.
Together with all those hoping for a healthy life and everyone standing beside them, the MEDIPAL Group will continue to light the way toward new treatments.