Published October 2022
| public
Journal Article
Effects of PB-TURSO on the transcriptional and metabolic landscape of sporadic ALS fibroblasts
Abstract
ALS is a rapidly progressive, fatal disorder caused by motor neuron degeneration, for which there is a great unmet therapeutic need. AMX0035, a combination of sodium phenylbutyrate (PB) and taurursodiol (TUDCA, TURSO), has shown promising results in early ALS clinical trials, but its mechanisms of action remain to be elucidated. Therefore, our goal was to obtain an unbiased landscape of the molecular effects of AMX0035 in ALS patient-derived cells.
Additional Information
We thank Amylyx Pharmaceuticals for providing the compounds used for this study. We thank Dr Hiroshi Mitsumoto (Columbia University) and the COSMOS initiative for providing the fibroblast and plasma samples utilized in this work. We acknowledge the contribution of the Weill Cornell Genomics Resource Core Facility and the Medicine Meyer Cancer Center Proteomics & Metabolomics Core Facility. This work was supported by funds from the NIH/NINDS (grants R35 NS122209 to GM and R21 NS104520 to HK and GM). Sponsored research agreement with Amylyx Pharmaceuticals.Additional details
- Eprint ID
- 117011
- Resolver ID
- CaltechAUTHORS:20220916-664913000
- R21 NS104520
- NIH
- R35 NS122209
- NIH
- Amylyx Pharmaceuticals
- Created
-
2022-10-29Created from EPrint's datestamp field
- Updated
-
2022-11-01Created from EPrint's last_modified field