@emilykmay My daughter is also non-verbal severe ASD. I am MD with BS in molecular biology . Dont like RFK but like the attention it brings to our kids. We are trying leucovorin - study published in Nature showing benefit for ASD. Watching NNZ-2591 trials from Neuren as well.
@adamfeuerstein You are a political science major with no background in science or journalism or statistics and it shows. You couldn't dismantle an IKEA bookshelf.
Neuren Pharmaceuticals announced succes in Phase 2 trial for Phelan-McDermid, a form of autism. The drug NNZ-2592 is an analog of trofinetide which worked in Rett Syndrome. Why not a trial in autism???!!! https://t.co/95ufi88teV
It's clearly outrageous to erase children with complex needs from the school photo.
But where is the outrage about children/adults with #profoundautism who are largely erased from public discourse, media representation, and even most scientific research?
https://t.co/TaW4HTzvHx
Today, during their quarterly financial call, @TayshaGTx shared updates and early interim data from the first two adult participants dosed with their investigational gene therapy TSHA-102 in the REVEAL Adult Study. Read the full press release here: https://t.co/ve7GMjyBf7
If approved, trofinetide will be the first-ever drug for the neurodevelopmental condition Rett syndrome. @acadiapharma is "eager" for 12 March, the day the FDA will issue its decision.
By @DrShaena.
@brimblem
https://t.co/3vsaqopHDX
BREAKING NEWS -
@NeurogeneInc just announced that the FDA approved their IND to launch a clinical trial for their #genetherapy treatment NGN-401. It will launch in the US in 2023 for pediatric females with #Rettsyndrome. Learn more here: https://t.co/EDZgUF9ivm
#HappyNewYear to families affected by #autism everywhere. Let's hope 2023 brings new impetus to research into treatments for disabling #ASD symptoms in order to help achieve a better quality of life for individuals with autism.
“Amongst all this heterogeneity and all these differences in autism, there might be some common driver,” says study investigator Smrithi Prem.
By @avaskham.
@biorxivpreprint: https://t.co/Zv9memtEwB
@david_sulzer@PeterCrino@Premsm2020
https://t.co/pe01nONhcF
Yet more evidence of mTOR's effect on ASD - unfortunately, rapamycin did not work in tuberous sclerosis
Autism subgroups converge on cell growth pathway https://t.co/aMcBrml8w5 via @Spectrum