The first podcast of 2022 discusses two papers with negative outcomes and explores the importance of sharing such results for patients and families and clinicians working with rare disease. Dr Nicoline Løkken discusses her work with resveratrol in mitochondrial myopathies (from 5m 18s) and Dr Arunabha Ghosh and Professor Brian Bigger talk about the use of genistein in Sanfilippo syndrome (from 10m 38s).
No effect of resveratrol in patients with mitochondrial myopathy: A cross-over randomized controlled trial
Nicoline Løkken, et al
https://doi.org/10.1002/jimd.12393
High dose genistein in Sanfilippo syndrome: A randomised controlled trial
Arunabha Ghosh, et al
https://doi.org/10.1002/jimd.12407