MyFSHD

"Good heavens, are you still trying to win? You've got an over developed sense of vengeance. It's going to get you into trouble someday." We remember Jenny Hasenjaeger and discuss research funding so that we don't let anyone else down.

"You know the Greeks didn't write obituaries. They only asked one question after a man died: 'Did he have passion?'. " Today we are joined by our CRISPR Goddess to discuss her new FSHD optimized gene therapy cassette that is stronger, safer, more compact and all around better than anything out there for use in FSHD and neuromuscular gene therapy approaches.

"Show me the money!" Brad our Angry Dad sits down with us and asks the questions on his mind about the Fulcrum results and others going forward.

"I always tell the truth, even when I lie." Today we are joined by our CRISPR Goddess and cover the Fulcrum Therapeutics REACH phase 3 clinical trial data release and discuss our journey with Fulcrum from the beginning. While very disappointing from several aspects, in the end a lot of benefit truly has been gained for the FSHD field and we are grateful for Fulcrum's contributions to help others going forward. And there are many others coming along, this is the beginning and not the end for FSHD therapeutics.

"There's a time for daring and there's a time for caution, and a wise man understands which is called for." Brad our Angry Dad is back with some questions for us on the Avidity FORTITUDE AOC 1020 phase 1/2 trial interim data report and some muscle building.

"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 phase 1/2 trial.

"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.

"We don't get a lot of things to really care about." So, you think you are interested in science, eh? Well, let's see how the sausage is made. Today we share our experience generating the FSHD-like minipig models, which will be key tools for testing and advancing better FSHD therapeutics and developing methods for building back your muscles.

"Come out to the coast, we'll get together, have a few laughs..." The holiday season we discuss the recent FDA approved CRISPR therapy for sickle cell disease and some dynamics of methylation.

"As I leave my competition respirator style, climb the ladder to success escalator style." Today we have the Jaegercast, but first we continue our discussion about apabetalone, a new candidate drug for FSHD.