Mom Feared Daughter Would Be ‘Locked in Her Body’—She Refused To Give Up [...] Nate Velasco2026-03-04T14:43:43-04:00 Read More
Meeting in the Middle: A Collaborative Approach to Tackling Rare Diseases [...] Nate Velasco2026-03-04T14:40:05-04:00 Read More
North Texas girl to become first patient to receive treatment for rare genetic disorder [...] Andrew Andrew Serrano2025-05-05T18:48:51-04:00 Read More
For the many and the few: bringing ASO therapies to both mainstream and nano-rare indications [...] Andrew Andrew Serrano2025-05-07T11:56:59-04:00 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
ASOs Drive Foundation’s Work to Develop Treatments for the World’s Rarest Diseases [...] Andrew Andrew Serrano2025-05-05T18:34:38-04:00 Read More
This lifesaving treatment was designed for one. Could it be the future of medical care? [...] Andrew Andrew Serrano2025-03-07T12:35:51-04:00 Read More
n-Lorem Foundation Bringing Real Hope To ‘n-of-1’ Patients [...] Andrew Andrew Serrano2024-08-13T21:15:06-04:00 Read More
