Girl with rare brain disorder thrives after gene therapy in San Diego [...] Nate Velasco2026-06-05T18:52:58-04:00 Read More
Mom Feared Daughter Would Be ‘Locked in Her Body’—She Refused To Give Up [...] Nate Velasco2026-05-11T12:03:35-04:00 Read More
Meeting in the Middle: A Collaborative Approach to Tackling Rare Diseases [...] Nate Velasco2026-06-05T18:10:17-04:00 Read More
North Texas girl to become first patient to receive treatment for rare genetic disorder [...] Andrew Andrew Serrano2026-05-11T12:04:11-04:00 Read More
For the many and the few: bringing ASO therapies to both mainstream and nano-rare indications [...] Andrew Andrew Serrano2026-05-11T12:01:58-04:00 Read More
Parents, doctors, medical researchers seek treatment for disease with no name [...] Andrew Andrew Serrano2026-03-04T14:35:57-04:00 Read More
A bespoke genetic therapy is helping Susannah. Can similar drugs be made at scale for other rare diseases? [...] Andrew Andrew Serrano2026-03-04T14:35:26-04:00 Read More
A teenager faced constant seizures. Could a drug developed just for him stop them? [...] Andrew Andrew Serrano2025-05-07T11:56:21-04:00 Read More
