Thiago Carvalho · @cyrilpedia
1688 followers · 8193 posts · Server qoto.org
Last Thursday, the FDA voted to approve Sarepta's microdystrophin gene therapy for #Duchenne muscular #dystrophy.
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy
This was not an easy call, made on the basis of a biomarker rather than efficacy data. I wrote about it last month for Nature Medicine.
#duchenne #dystrophy #genetherapy #drugdiscovery #drugdevelopment
FSHD News · @FSHD
7 followers · 15 posts · Server home.social
🇪🇺🇮🇹
📰🧬#FSHD #Muscular #dystrophy, the test that makes the diagnosis in 72 hours
Gerion · @ginslo
31 followers · 150 posts · Server mastodon.lolWorking with one eye as I recover from my cornea transplant. The gas bubble in my eye is helping to adhere and graft the donor tissue. Laying flat with eyes up for several hours per day allows the bubble to be centered, providing full coverage of the donor tissue. Vision in the new eye is extremely blurry due to the bubble but I see light and am optimistic for when the gas dissipates.
#surgery, #endothelial, #keratoplasty, #cornea, #transplant, #DMEK, #fuchs, #dystrophy, #ophthalmology
#surgery #endothelial #keratoplasty #cornea #transplant #dmek #Fuchs #dystrophy #ophthalmology
Gerion · @ginslo
31 followers · 150 posts · Server mastodon.lolGoing uner the knife tomorrow morning for a cornea transplant to treat Fuchs' Dystrophy. Grateful for modern medicine.
#surgery, #endothelial, #keratoplasty, #cornea, #transplant, #DMEK, #fuchs, #dystrophy, #ophthalmology
#surgery #endothelial #keratoplasty #cornea #transplant #dmek #Fuchs #dystrophy #ophthalmology