Activities Report / InformationREPORT & INFORMATION
2024.02.29
Activity report
- #Lysosomal storage disease
- #Participation report
- #Patient association
- #Mucopolysaccharidosis type III
20th Annual WORLDSymposiumTM 2024 Report
February 4 to 9, 2024- MEDIPAL HOLDINGS CORPORATION (MEDIPAL) participated in WORLDSymposiumTM 2024 held in San Diego, USA.
WORLDSymposiumTM is an international conference held annually in the US to report researchers, physicians, and patient groups on the latest advances in research on the rare disease, lysosomal storage diseases (LSD) (https://worldsymposia.org/).
At this conference, over 100 platform presentations, 350 poster presentations, and booths of approximately 40 companies and patient organizations related to LSD attracted attendees from more than 60 countries.
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MEDIPAL launched a new initiative in the field of ultra-rare diseases in 2022. This is the second time to participate in WORLDSymposiumTM (2023 report).
At the conference, we visited the other companies’ booths, attended platform presentations, and looked at poster presentations regarding the latest research. We gained many insights from various presentations, including new challenges in existing treatments, the current situation of newborn screening in each country, and the current situation of enzyme replacement therapy. In particular, there were many presentations on research related to gene therapy, which showed the progression of this treatment.
On the day of the conference, JCR Pharmaceuticals Co., Ltd. and MEDIPAL held some meetings with LSD patients’ families. We would like to show some comments from parents as follows.
【Comments from patient’s family】
* To avoid identifying individuals, all individual disease names are unified as “LSD.”
- When the doctor told us the name of the disease, it was like the end of the world. We didn’t know the words before. We never heard about the disease.
- The disease of my child is progressing so fast that we don’t have enough time. But we don’t want to give up.
- The first time of heard the name of this disease, we knew nothing about LSD. We want as many people as possible to know about LSD.
- My wife searched on Social Networking Services for symptoms such as speech delay, a slightly large head, and a very hairy back. She found a story about a mother having a child with LSD. Then we went to the pediatrics to test our child. As a result, our child was diagnosed with LSD.
- It was really hard for me when our child was diagnosed. Currently, my child can communicate and has social skills, but we are very anxious about how the disease will happen in the future.
We would sincerely listen to the voices of patients and their families. We will continue to strengthen our efforts against our activities deepen our understanding of ultra-rare diseases, and contribute to patients and their families suffering from diseases for which there is no treatment yet.
WORLDSymposiumTM 2025 is scheduled to be held in San Diego, US, from February 3 to 7. We are scheduled to participate in the conference.
