2024.02.29

Activity report

  • #Lysosomal storage disease
  • #Participation report

The 28th Annual Meeting of the Japanese Society for Lysosomal Storage Diseases Report

On November 10, 2023, The 28th Annual Meeting of the Japanese Society for Lysosomal Storage Diseases was held at The Jikei University School of Medicine. This aims to foster research on the pathogenesis, diagnosis, and treatment of lysosomal storage diseases, leading to early diagnosis and treatment.
In 2021 and 2022, the meeting was held using the web, it’s been four years that only in-person meetings. The contents of this meeting included many presentations on Fabry disease, Gaucher disease, Pompe disease, and Mucopolysaccharidoses.
In addition, this meeting is opened to the general as well as researchers and companies.

Points

  • The benefits and challenges of Enzyme replacement therapy, Hematopoietic stem cell transplantation, Chemical chaperone therapy, Substrate reduction therapy, and Gene therapy were presented.
  • Patient Reported Outcome, one of the methods to evaluate the effectiveness of the medication, has attracted attention in recent years and it was presented that the voices of patients and caregivers are also important in clinical trials.
  • It was introduced that COVID-19 has had a negative impact on the continuation of treatment for some patients because of restricting hospital visits to prevent the spread of the infection.
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