Rapamycin and Pharmacogenomics in Niemann-Pick C
About this episode
Dr Andrés Klein discusses the implications of pharmacogenomics in the treatment of Niemann-Pick C with a focus on rapamycin.
A study of rapamycin in Niemann-Pick C raises an important question: what if the success of a treatment depends on a patient's wider genetic background? Dr Andrés Klein discusses pharmacogenomics, modifier genes and why precision medicine may need to go far beyond making the diagnosis. A Rapamycin Pharmacogenomic Approach for the Childhood Dementia Niemann-Pick C Benjamín Szenfeld, et al https://doi.org/10.1002/jimd.70214
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