Breakthrough Gene Therapy For Butterfly Children’s Disease

A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research

In a landmark development for rare disease treatment, the U.S. Food and Drug Administration (FDA) has approved ZEVASKYN, the first autologous, cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), a…