Articles & PodcastsThe new era of individualized medicine requires a ‘genetic surgery’ systemJulia Vitarello ( STAT ) May 22, 2025 In 2018, my daughter, Mila, became the first person in the world to receive a medicine designed for...
Articles & Podcasts'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis
Articles & PodcastsN=1 Collaborative Welcomes Hugh Hempel as Executive Director to Lead the Next Phase of Growth
Articles & PodcastsBuilding a Bridge Between Rare Disease Patients and the Technologies that Could Help Them
Articles & PodcastsCan AI build custom ‘n-of-1’ drugs faster? Maybe not yet, new case study suggests
Articles & PodcastsAccelerated approval will be 'the norm' for gene therapies, FDA's Peter Marks saysZachary Brennan (Endpoints News) February 27, 2027 The FDA will increasingly use accelerated approval for gene therapies aimed at rare...
Articles & PodcastsFour Keys To Advancing Antisense Oligonucleotides For Personalized MedicineDemaris Mills (Forbes) January 12, 2024 The 2023 Oligonucleotide Therapeutics Society meeting drew 800 attendees to Barcelona, a clear...
Articles & PodcastsThis lifesaving treatment was designed for one. Could it be the future of medical care?
Articles & PodcastsOxford-Harrington Rare Disease Centre named first global initiative for children with rare disease
Articles & PodcastsPilot launched to support children with rare conditions to access personalised therapiesGenomics England November 22, 2023 New programme announced to develop a pathway for children with rare conditions to access therapies...
Articles & PodcastsWhat We Owe Terry Horgan: Reflections from Providers, Family, and ScientistsOctober 12, 2023 I first saw Terry Horgan as a patient. I was filling in as the pediatric pulmonary specialist in the UMass-Duchenne...
