Reverse transcriptase inhibitors in Aicardi–Goutières syndrome: A crossover clinical trial
There is insufficient evidence that ABC or 3TC is either effective or ineffective in reducing type I IFN signalling in AGS over 6 weeks. The effect of ABC + 3TC + AZT at 3 weeks supports data from a previous clinical trial of the effect of ABC + 3TC + AZT in reducing type I IFN signalling, although there was insufficient evidence of an effect at 6 weeks. Time to local research and development (R&D) approval, and to sponsor authorization after R&D approval, severely limited patient recruitment.
Authors
- Yanick Joseph Crow (ORCID: https://orcid.org/0000-0001-7211-7564)
- Claire Battison (ORCID: https://orcid.org/0000-0001-9549-4522)
- Richard A. Parker (ORCID: https://orcid.org/0000-0003-4888-8670)
- Titouan Kennel (ORCID: https://orcid.org/0000-0002-2783-2945)
- Tracy A. Briggs (ORCID: https://orcid.org/0000-0001-5208-2691)
- Despina Eleftheriou (ORCID: https://orcid.org/0000-0002-5312-2269)
- Amitav Parida
- A E B Giddings
Institutions
- Inserm (FR)
- Manchester Academic Health Science Centre (GB)
- Great Ormond Street Hospital (GB)
- Great Ormond Street Hospital for Children NHS Foundation Trust (GB)
- University of Manchester (GB)
- St Mary's Hospital (GB)
- Birmingham Children's Hospital (GB)
- Institute for the Study of Knowledge Management in Education (US)
- Genomics (United Kingdom) (GB)
- Birmingham Women’s and Children’s NHS Foundation Trust (GB)
- Institute of Genetics and Cancer (GB)
- Institut des Maladies Génétiques Imagine (FR)
- St. Mary's Hospital (GB)
- University College London (GB)
- University of Edinburgh (GB)
Publication Details
- Journal
- Developmental Medicine & Child Neurology
- Published
- 2024-12-04
- DOI
- https://doi.org/10.1111/dmcn.16199
- Citations
- 5
- Primary Topic
- interferon and immune responses
- Type
- article
- Field-Weighted Citation Impact
- 1.20
Funders
- Medical Research Council