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.

Explore the reality of Gene Therapy in rare conditions. Is it a genetic fix or false hope?

Tragic deaths in Sarepta’s gene therapy trials have deeply impacted the rare disease community, raising concerns about liver risks, FDA responses, and treatment access.

In a groundbreaking first, doctors developed a custom gene- editing therapy in just seven months to treat a baby with a deadly and ultra-rare metabolic disorder.

New research on "jumping genes" could lead to safer, more precise gene-editing techniques.

Retinitis Pigmentosa (RP) is a group of rare, inherited eye disorders leading to progressive vision loss.

Explore the FDA's extension of the priority review period for Kresladi, a groundbreaking gene therapy for severe LAD-1

Explore the FDA's groundbreaking approval of CASGEVY™ the treatment of transfusion-dependent beta-thalassemia (TDT).