February 21 to 26, 2023- Four members from MEDIPAL HOLDINGS CORPORATION (MEDIPAL) participated in WORLDSymposium^TM 2023 held in Orlando, USA.

WORLDSymposium^TM is an international conference held annually in the US to report on the latest advances in clinical trials and research related to lysosomal storage diseases to people involved with lysosomal storage diseases, including researchers, physicians, and patient groups (https://worldsymposia.org/). Approximately 3,000 people from around the world participated in this year's event.

In 2022, we launched a new initiative in the field of ultra-rare diseases with JCR Pharmaceuticals and participated in WORLDSymposium^TM 2023 for the first time.

On that day, we held a meeting with families of patients with fucosidosis^*1 and neuronal ceroid lipofuscinosis (NCL)^*2 together with JCR Pharmaceuticals and listened to their comments, which included: “We are grateful your development of a treatment in a disease area that has no treatment,” “We have high expectations for MEDIPAL and JCR,” and “We are willing to cooperate in MEDIPAL's activities.” MEDIPAL intends to promote activities that will help increase understanding of such diseases and raise their awareness for patients who are waiting for a treatment.

In addition to visiting the booths of related organizations, participants listened to oral and poster presentations on the latest research and gained new knowledge.

JCR Pharmaceuticals presented a poster about a survey of patients with fucosidosis.
We feel that these reports will help deepen the understanding of fucosidosis and gradually increase awareness of the disease.

Participating in WORLDSymposium^TM 2023 was a great opportunity to deepen our knowledge of new research, medication development, and diseases. We will continue to strengthen our efforts against ultra-rare diseases in cooperation with affiliated companies.

The WORLDSymposium^TM 2024 is scheduled for February 4 to 9, 2024, in San Diego, USA., and some of our employees are scheduled to participate there.

*1：

About fucosidosis

*2：

About Neuronal ceroid lipofuscinosis (NCL)
NCL is a lysosomal storage disease that is also referred to as Batten disease. It is a neurodegenerative genetic disease and inherited in an autosomal recessive pattern. NCL is mainly characterized by progressive motor dysfunction, convulsions, and vision impairment. In general, the prognosis is extremely poor when the disease first appears in childhood. NCL can be classified into CLN1 to CLN14 based on the causative gene mutation.
CLN1 is caused by low activity of an enzyme that removes palmitic acid (palmitoyl-protein thioesterase 1: PPT1). It is classified into four types based on the age of onset: infantile, late infantile, juvenile, and adult. There is no standard therapy available for this disease.
CLN2 is caused by low activity in a proteolytic enzyme (tripeptidyl peptidase 1: TPP1). It is classified into infantile and juvenile forms. TPP1 enzyme replacement therapy by intracerebroventricular administration has been approved in Europe and the U.S. in 2017 and Japan in 2019 as a standard of care for this disease.