Rare Disease Day and Accountability
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Description
"Sorry, Vern. I guess a more experienced shopper could have gotten more for your seven cents."  Rare disease day is the last day of February, which got us thinking about what really needs to be done to get over the line.  We also answer listener questions about animal models and the Avidity, myostatin inhibition, and cell therapy trials. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message
More Episodes
"Whatchyou talkin' 'bout Willis?" In a field where every press release is met with unsubstantiated hoopla and proclamations of BREAKTHROUGH! to fire you up to shake you down, we finally have some real news. We provide our evaluation of Avidity's public interim report on their FORTITUDE AOC-1020...
Published 06/14/24
Published 06/14/24
"You're messing with the wrong guy!!!!" We expand a bit more on the utility of the FSHD-like pig model and then discuss the science behind and implications of p38 inhibition for FSHD. --- Send in a voice message: https://podcasters.spotify.com/pod/show/peter-l-jones/message
Published 06/01/24