O’Neill joins other rare disease advocates for meeting with FDA Commissioner, Directors

April 18, 2023

Last week, Cure Sanfilippo Foundation Chief Science Officer and Co-Founder Cara O’Neill was privileged to be among a group of fellow ultra rare disease advocates meeting with the FDA Commissioner Robert Califf, MD, and Center Directors.

The group presented the urgent challenges and barriers to drug development that are directly impacting the process toward approval of effective therapies for Sanfilippo Syndrome and other similarly-devastating ultra rare genetic diseases.

“It says something important that FDA leadership took time to meet with us and hear the scientific and disease communities’ concerns, something which we truly appreciate,” said O’Neill.

“Our hope is that through collaboration, communication and action around the critical issues raised, we will work together to accelerate a lifeline to effective therapies for children who are in dire need.”

Sanfilippo Syndrome is a disease like Alzheimer’s in children. This terminal, degenerative, rare disease causes children to lose all the skills they’ve gained, suffer seizures and movement disorders, experience pain and suffering, dementia, and then die usually in their teen years.

Currently there is no FDA-approved treatment or cure for Sanfilippo Syndrome.

Cure Sanfilippo Foundation is a 501(c)3 nonprofit dedicated to advocating and funding research to bring about treatments and/or a cure for Sanfilippo Syndrome. Cure Sanfilippo has funded 35+ research grants around the world which has helped three clinical trials get underway, to date, treating children with Sanfilippo Syndrome. The Foundation creates and explores innovative and breakthrough research to ensure multiple promising paths are being explored in the effort to save children and better their quality of life.

Related Posts