"...and by the time they figure out what went wrong, we'll be sitting on a beach, earning twenty percent." This is part 2 of our podcast series covering the important factors in successful gene therapy and introduces the knowledge gained from other neuromuscular diseases and how it can be applied to positively impact gene therapy in FSHD. In part 2, we focus on viral dosing and how it affects therapeutic biodistribution and expression. In addition, we reveal what is known about variable durability of transgene expression and what to look for to predict a durable, or non-durable, therapeutic. Check out the MyFSHD Youtube for the music, as well.

"My friends are toys. I make them. It's a hobby. I'm a genetic designer." This is a two part podcast covering the important factors is successful gene therapy and introduces the knowledge gained from other neuromuscular diseases and how it can be used to positively impact gene therapy in FSHD. In part 1, we focus on AAV serotypes, transgene constructs, immune responses, and delivery. We look into DMD gene therapy and how that can impact the current FSHD gene therapy programs. Check us out on the MyFSHD Youtube channel as well.

"You were seeking some information of some kind?" Continuing our discussion with our young FSHD advocates, asking and answering some questions.

"And that would tell you what is obvious right now? That avoiding apple skins and pizza has no effect on this brutal disease?" Today we discuss the Cure FSHD for All (go to: www.curefshdforall.com) initiative for inclusion of FSHDers under 18 and those with limited mobility into clinical trials and for access to developing therapies.

"You're gonna need a bigger boat." MyFSHD is our summer blockbuster passion project. We discuss how to get it done, whatever your "it" is, in the FSHD space, as well as our trip to Shanghai and Hanoi to continue our efforts to help FSHDers around the world.

"I'll have what she's having." We'll take you through and discuss the recent release of Avidity Biosciences topline results from the phase 1/2 FORTITUDE trial and the preliminary data from the biomarker cohort extension.

"I gotta know what a 5 dollar shake tastes like." We continue our conversation with Tamara Gottlieb, who shares her years of experience helping to keep her family above the FSHD curve and improve quality of life. It's a lot of work, but it can help. Part 2 of 2. Tamara is one of the leaders (along with Emily Ca and Wayne Nesbit) of the "FSHD - supplements, nutrition, and peer support" Facebook gropou, so check it out for more information.

"You are what you eat, and I'm freaking delicious!" Our guest today, Tamara Gottlieb, is the driving force for our First Family of FSHD Fitness, Nutrition, and Lifestyle, and she shares with us what she has learned over her years of experience helping keep her family above the FSHD curve and improve quality of life. It's a lot of work, but it can help. Part 1 of 2. Tamara (along with Emily Ca and Wayne Nesbit) is one of the leaders of the "FSHD - supplements, nutrition, and peer support" Facebook group, so check it out for more information.