Cure Sanfilippo Foundation Presenting at WORLDSymposium 2021

The Cure Sanfilippo Foundation will be among those presenting posters at the WORLDSymposium 2021 conference on lysosomal storage disorders such as Sanfilippo syndrome, held virtually Feb. 8–12.

“This is the conference where most of the scientists and researchers in the field of lysosomal storage diseases come together to share results, collaborate, and advocate for this disease group, which includes Sanfilippo Syndrome,” Cara O’Neill, MD, chief science officer for the Cure Sanfilippo Foundation, said in a press release.

Registration for this year’s conference is free for all lysosomal disease patients and their immediate family members.

Keynote speakers include Peter Marks, the director of the Center for Biologics Evaluation and Research at the U.S. Food and Drug Administration, and Michael Osterholm, the director of the Center for Infectious Disease Research and Policy.

This year’s conference will feature more than 330 scientific abstracts delivered at five poster sessions, separated broadly by topic. These topics are basic science, translational research, COVID-19 and clinical trials, contemporary forums, and late-breaking abstracts.

Posters to be presented on Sanfilippo syndrome will cover: the ongoing progress made in three gene therapy trials; two prospective studies of the disorder’s natural history; enzyme replacement therapy; and a study of immune system defects in certain patients that was reversed by the anti-inflammatory medication anakinra, which is used often to treat rheumatoid arthritis.

Cure Sanfilippo has two of the poster presentations and O’Neill will give a talk titled “Patient’s Perspective on Experimental Therapies,” during the conference’s Emerging Trends session, held on Sunday from 10 a.m. to 2 p.m. EST.

These 20-minute talks review the past year’s advances, summarizing the latest research trends, new knowledge, and other discoveries. Other subjects include newborn screening, several case studies, the SARS-CoV-2 virus, and the impact and management of COVID-19.

Talks from that session will remain available to watch on-demand until March 15.

Cure Sanfilippo will maintain a virtual booth in the exhibit hall and members from the foundation will attend talks and take notes for those who cannot attend themselves.

“We are making the best of the virtual setup this year and looking forward to connecting with researchers, scientists patient families, and regulatory leaders,” said Glenn O’Neill, president of Cure Sanfilippo Foundation.

The WORLDSymposium encourages use of social media throughout the conference, but asks that attendees not share specific data or slides from the talks, or to use the symposium’s Twitter and Facebook platforms to comment on medical, legal, or litigious matters.

More information is available here.