Replay Link: https://accountingdepartment.wistia.com/s/ir33y49byi7i6ic
The Natural History Study of AHC which was published recently has major implications for the care of AHC champions and for anticipated clinical trials. This study highlights, among other things, many prognostic factors that help chart the clinical course of specific patients, that can help families know what to expect and thus better care for these champions. You can view the study here: https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70037
Mohamad A. Mikati MD, FAS, Davison Professor of Pediatrics, Professor of Neurology, Duke University who has received multiple awards and is listed by research.com as one of the ranked Best Scholar Neuroscientists nationally and worldwide. He has published 350 scientific articles, 55 are about AHC/ATP1A3.
He received his MD at the American University of Beirut then trained in Neurology and Neurophysiology at Harvard.
His basic science and clinical research concentrated on epilepsy and on Alternating Hemiplegia of Childhood (AHC). In AHC, he was the first to demonstrate, in 1992, familial occurrence of AHC, thus proving that it is a genetic disease.
He also was the first to develop an AHC-USA Registry at Harvard and to report, in 2000, based on that registry, on the totality of AHC manifestations and long-term outcome. Subsequently, he was one of 2 senior and corresponding authors, contributed equally, of the Nature Genetics article that, in 2012, demonstrated that AHC is caused by ATP1A3 mutations.
Then, in 2015 and 2018, he generated mouse models of the two most common AHC mutations, established that they faithfully reflect human AHC manifestations and characterized their neuronal electrophysiology.
He led the team that was the first to demonstrate that gene therapy increases survival and ameliorates several manifestations in AHC mice.
He also established, at Duke, the leading center for care and clinical research for patients with AHC in the USA and generated a series of publications (2015-2026) regarding the manifestations and therapies of AHC.
He co-founded, led and collaborated with other members of the International AHC Consortium (IAHCRC) on clinical studies of AHC including the international natural history study, in 2025.
He continues to pursue active clinical and basic research aiming at curing AHC.
