The last line is an IMPORTANT READ ....FDA gives orphan drug status to Avidity’s rare muscular dystrophy treatment

pretty sure theres already a natural treatment out there already. my mom has it the gene and has asymptomatic form of muscular dystrophy at 60 which is bizarre as fuck until I was asking around to other people and apparently it is possible to go into your 80s with this without a clue. why dont they study these people more? we've sent my mothers spit sample to jones and he knows hes been studying them for biomarkers. Its kind of like with ALS reversals or people who dont get AIDS. ALS has a clinic for people who somehow have reversed this disease and theres a guy studying them. So you need something similar for fshd because there are many asymptomatic people with it. ive seen some small studies but theyre sparse and not really looked into it? its like you have this untapped population.

/r/FSHD Thread Link - pmlive.com