Global Foundation For Peroxisomal Disorders

Research and medicinethe gfpd continues to maintain and grow a strong committee of physicians and scientists as part of our msab. These individuals currently comprise researchers, clinicians, and experts from north america and europe who treat and assist peroxisomal patients through clinical medicine and research. Primary research partners include mcgill university, baylor college of medicine, university of southern california, university of amsterdam, the kennedy krieger institute, and montclair state university. Additionally, the gfpd has clinical advisors from a number of children's hospitals throughout north america. To further gfpd awareness and networking, representatives held meetings with the national organization for rare disorders, global genes, the american college of medical genetics, the notre dame rare disease symposium, the world orphan drug congress, and the american association for the study of liver disease.

Webinar and virtual event seriesthe gfpd is the premier resource for information, clinical direction, treatment guidelines, networking, medical linkage, and online learning for patients, families, caregivers, clinicians, physicians, and scientific professionals with a range of familiarity and expertise in peroxisomal disorders and their co-morbidities. One of the gfpd's flagship programs is its webinar and virtual event series. This program provides year-round events for patients, families, and professionals. The program is offered at no cost to patients, families, and caregivers, and provides crucial support, education, advocacy tools, and resources. The virtual environment enables more flexibility in scheduling, recording, and disseminating information to patients, families, and caregivers, which increases access to our community. The gfpd webinar and virtual event series provides opportunities for consistent and direct engagement with the community we serve, and shapes our strategy for improving webinars, other family programs, and research collaborations. In october, a first-time event proved the perfect example of the impact of virtual webinars and events. A "relax and relate" virtual gathering was hosted for our extremely small adult patient community, in which eight individuals met, many for the first time. The patients shared that they hoped to gain a connection to others, to not feel alone," to learn from one another about their experiences, and what has and has not helped with symptom management. There was unanimous interest in participating in future similar gatherings, as well as having themed topics and guest presenters on various subjects. Overall, in 2024, the gfpd hosted 28 events and had an average net promoter score of 89.2.