Researchers have discovered a potential “window” whereby babies born with inherited diseases may be treated with gene therapy sent directly into their circulatory systems. The window consists of circulating stem…
An article titled “Accelerated Approval as the New Norm in Gene Therapy for Rare Diseases” from DIA Global Forum digital magazine’s May 2025 issue explores the evolving landscape of regulatory…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Drs. Julie Gerberding…
Have you ever wondered how a rare disease can take control of a person’s life? The deteriorating health, being unable to receive an accurate diagnosis, and more importantly finding an…
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