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Appreciate this presentation. Like others I think it might be helpful in the future to have separate webinars for AL and TTR. Have you any information on a new drug for TTR: SOM0226, under development in Spain by a company called SOMBiotech? They claim that it has potential to clear existing amyloid deposits from the heart. The decision tree for TTR treatment options was of particular interest. I am enrolled in the Tafamidis trial, but of the options currently available Doxy/URSO seems most probable to slow progression. What is your opinion? Has any research focused on why WT TTR primarily effects men? It seems that might somehow shed light on the causes of this condition. Thanks to all involved.
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Yes, for future we will consider separate webinars. I am not aware of the spanish drug, will check into it. We really don't know which treatment option will be best and that's why we need the clinical trials. Many examples of clinicians and scientists being surprised one way or the other about results. The male predominance is fascinating, although we do see some women with WT ATTR. It may be that women do get the disease and it "looks different" so we don't recognize it. There may be hormonal influences. This is an area of investigation and I agree that more research into gender differences may help us understand the cause and help develop treatment options.