San Diego family helps fund gene therapy approved for rare disease

KPBS reported that a family affected by Sanfilippo syndrome in San Diego raised millions of dollars to support research on the rare genetic disorder.

Their fundraising efforts helped advance the development of a gene therapy, which has now received approval from the Food and Drug Administration (FDA).

Sanfilippo syndrome is a rare disease, and the newly approved gene therapy represents a critical advancement in treatment options for patients.

The family’s contribution highlights the important role local advocacy and fundraising can play in accelerating medical research and approvals.

This development brings hope to families affected by Sanfilippo syndrome in the San Diego area and beyond, as gene therapy becomes a viable treatment option.