Four Keys To Advancing Antisense Oligonucleotides For Personalized Medicine
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Demaris Mills (Forbes) January 12, 2024 The 2023 Oligonucleotide Therapeutics Society meeting drew 800 attendees to Barcelona, a clear...
Connecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting
Monday, March 4, 2024 12:30 pm US EST From an initial email request or conversation at a meeting… to (for some) the possibility of...
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Patient Partners: Harnessing synergies to pursue ASOs as part of the AHC research portfolio
Monday January 29, 2024 12:30 PM US EST Mary will share her son Henry's case and describe her efforts to develop an n=1 ASO for him. Nina...
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Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders
Marlen C. Lauffer, Willeke van Roon-Mom, Annemieke Aartsma-Rus & N=1 Collaborative (Communications Medicine) January 5, 2024 Abstract...
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N1C Patient Identification Workgroup: Compilation of Useful Tools
Click below for download: Disclaimer: This sample document is the work product of the N=1 Collaborative. This document is intended to...
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Parents of Pioneers: Real experiences in individualized medicines today
Monday, December 18, 2023 at 12:00 pm ES EST Julia Vitarello, Mila's Mom, Mila's Miracle Foundation Julia founded Mila's Miracle...
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This lifesaving treatment was designed for one. Could it be the future of medical care?
Karen Weintraub (USA Today) November 26, 2023 Imagine you or your child gets a diagnosis so rare no one else on Earth is known to have...
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Oxford-Harrington Rare Disease Centre named first global initiative for children with rare disease
University of Oxford November 24, 2023 In the autumn statement on Wednesday 22 November, the government announced its support for the...
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