Articles & Podcasts‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
Articles & PodcastsA young girl’s custom gene therapy hints at a framework for tailored rare disease treatments
Articles & PodcastsHopes for Antisense Oligonucleotides Remain Strong Despite Severe Adverse Events in Trials
Articles & PodcastsDrug tailored to one girl with brain disease paves way for similar customized treatments
Articles & PodcastsThis girl’s dramatic story shows hyper-personalized medicine is possible—and costly
Articles & PodcastsAfter a bespoke therapy rescues a young girl, the FDA considers advance of individualized treatments
Articles & PodcastsWhen ‘right to try’ isn’t enough: Congress wants a single ALS patient to get a therapy never tested
