Seminar SeriesConnecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting
Seminar SeriesPatient Partners: Harnessing synergies to pursue ASOs as part of the AHC research portfolio
PublicationsPossibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders
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...
