I recently attended the 2026 WORLDSymposium in San Diego, which brings together research centers, clinicians, advocacy groups, patients, and pharmaceutical companies – all of whom are stakeholders in the lysosomal dysfunction disorders (LSD) space.
The agenda was very packed and included lectures, poster presentations, awards programs, mentoring sessions, and time to meet with colleagues, form new connections, and to decompress. Gaucher disease, as the “flagship” lysosomal disorder, was addressed in numerous talks and posters. In fact, it is no exaggeration that all work and progress done on other LSD disorders has been predicated on the original work done which revealed the compromised enzyme in Gaucher by Dr. Roscoe Brady (NIH) in the 1960s. Hence, each year one individual whose contributions are judged to be of major significance is given an award in Dr. Brady’s memory and honor.
The presentations were divided into categories. On Tuesday, lectures were focused on basic science efforts; on Wednesday, lectures were given on translational work, which is what takes knowledge gained from work on cells to work on animals.
On Thursday and Friday, the lectures discussed clinical trials. The poster presentations followed the same path and allowed many academic pursuits to be showcased, as time for lectures is limited.
This flow of information allows us to follow the process of discovery and implementation.
There were talks on all types of Gaucher disease: 1, 2, and 3. These talks included work on improving enzyme delivery methods, “upgraded” enzymes and delivery methods, various genetic interventions, biomarkers for diagnosis for following treatment responses both in clinical trials and in real world patient responses. Centers worldwide presented their systems for diagnosis and patient treatment.
I took the opportunity to have photos taken of myself and only a few of the many presenters on Gaucher disease.
It was thoroughly enjoyable, intense, and beneficial to me to personally meet so many people worldwide working on improving the lives of those affected by Gaucher.
When I look back at the 42 years since I co-founded NGF, I am truly amazed and feel so blessed at the explosive nature of today’s Gaucher space. It is a constant effort to keep abreast of the vast number of centers working in our field, and the yearly WORLD conference continues to be an incredible encapsulation of the efforts of the many wonderful human beings behind the scientific and clinical results that have had such major worldwide impact.
