Family Boosts Gene Therapy Research for Sanfilippo Syndrome

A family affected by Sanfilippo syndrome has raised millions of dollars to support research for a new gene therapy, which has just been approved by the Food and Drug Administration. This achievement is a significant step forward in the fight against this rare disease, showing how community support can lead to medical advancements. The family's efforts highlight the importance of research and innovation in healthcare, inspiring others to contribute to causes that can change lives.

Based on reporting from NPR. Read the original story →