Lafora disease is a rare, fatal genetic disorder affecting the brain, with limited treatment options and severe symptoms.

Epidermolysis Bullosa, also colloquially known as butterfly skin disease, a rare genetic condition that makes your skin very ...

Researchers have found a likely cure for a rare genetic disease – and saved the lives of nine children in a clinical trial in ...

Hundreds of people gathered in New Hartford Saturday morning for the fourth Annual Grateful for Graham 5K Run and Walk.

Patients with severe dystrophic epidermolysis bullosa, or EB, have skin so fragile, the slightest touch can lead to ...

In mice with human Merkel cell carcinoma, pyrvinium pamoate significantly reduced tumor growth, potentially offering a new ...

An investigational gene therapy has successfully restored immune function in all nine children treated with the rare and life ...

The U.S. Food and Drug Administration (FDA) has approved Abeona Therapeutics Inc.’s ZEVASKYN gene-modified cellular sheets.

Two years after receiving an investigational gene therapy as part of a clinical trial co-led by UCLA’s Dr. Donald Kohn, nine children born with a life-threatening immune disorder are living free of ...

Barely a year after the U.S. FDA shackled Abeona Therapeutics Inc.’s cell-based gene therapy with a complete response letter, the agency has approved it for treating a rare and genetic skin disease.

In a phase 3 trial, 81% of wounds treated with autologous cell sheet-based gene therapy showed ≥ 50% healing at 6 months, ...

ZEVASKYN is expected to be available beginning in the third quarter of 2025 through ZEVASKYN Qualified Treatment Centers, ...