Parents, doctors, medical researchers seek treatment for disease with no name [...] Andrew Andrew Serrano2025-03-07T12:39:13-04:00 Read More
A bespoke genetic therapy is helping Susannah. Can similar drugs be made at scale for other rare diseases? [...] Andrew Andrew Serrano2024-08-13T21:17:30-04:00 Read More
A teenager faced constant seizures. Could a drug developed just for him stop them? [...] Andrew Andrew Serrano2024-07-17T22:25:21-04:00 Read More
ASOs Drive Foundation’s Work to Develop Treatments for the World’s Rarest Diseases [...] Andrew Andrew Serrano2024-07-17T23:25:10-04:00 Read More
