For decades, Huntington’s disease had no treatment that could alter its course. The new AMT 130 gene therapy trial shows promise in slowing disease progression, marking a historic milestone for patients, families, and the broader rare disease community.

Rare diseases don’t just affect health—they place overwhelming financial and emotional strain on patients and caregivers. From costly treatments to lost wages, this article uncovers the hidden burden of rare diseases and why systemic policy change is critical.

Dialysis can be different for people living with rare kidney disorders. Learn what to expect, how to navigate challenges, and find hope for the future.

This article breaks down what the ODA is, why it matters, the challenges that remain, and what patients and caregivers need to know to advocate for continued progress in rare disease care.