Guidance on communication and informed consent with patients and their families for experimental individualized treatments
Annemieke Aartsma-Rus, Claudio M. de Gusmao, Scott Demarest, Matias Wagner, Georg Marckmann, Charlotte Kogel, Roseline Favresse, Rebecca Schuele, Matthis Synofzik, Holm Graessner, Lynn Bush, Timothy W. Yu · 2026
Read Guidance on communication and informed consent with patients and their families for experimental individualized treatments by Annemieke Aartsma-Rus, Claudio M. de Gusmao, Scott Demarest, Matias Wagner, Georg Marckmann, Charlotte Kogel, Roseline Favresse, Rebecca Schuele, Matthis Synofzik, Holm Graessner, Lynn Bush, Timothy W. Yu (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13277676.txt.
Description
Annemieke Aartsma-Rus and colleagues offer 2026 guidance on consent talks for experimental one-person genetic treatments. New tools can pinpoint rare genetic diseases even when no approved treatment exists. For a few patients, a bespoke therapy can be built to correct their exact defect.
Standard trial consent covers much, yet these cases add deep uncertainty about benefit and harm. Families must weigh biological plausibility and early lab data against a serious, often fatal, condition. The authors draw on shared experience with individual antisense oligonucleotide treatments. They set out how to discuss risks, data sharing, and the right to withdraw without losing ordinary care. The guidance is presented as a living starting point.
Catalog record
| CREATOR | Annemieke Aartsma-Rus, Claudio M. de Gusmao, Scott Demarest, Matias Wagner, Georg Marckmann, Charlotte Kogel, Roseline Favresse, Rebecca Schuele, Matthis Synofzik, Holm Graessner, Lynn Bush, Timothy W. Yu |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 28 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |