Nonprofit research advocacy org for conditions caused by dysfunctional RAS/MAPK signaling. #rasopathies #raredisease
by Stronach in Pittsburgh
RASopathies Network
Loading...
This is a progressive fatal disease.
I hope this leads to real change for the DMD community.
Relevant to #RASopathies
Not uncommon for ppl w these syndromes to have Chiari malformation requiring surgery.
π§
β¨ Looking forward to hearing from Paul Trainor at #2026SDB in Las Vegas! Paulβs work at the Stowers Institute on neural crest development in mammals (sometimes fish and chameleons) has helped bring attention to understudied craniofacial conditions. π¬ #devbio #Stowers25
Super interesting work on the role of Myo1a in snail L/R asymmetry!
An ancient polymorphism in myosin I a/b determines the left-
right asymmetry of Japanese snails
www.biorxiv.org/content/10.6...
Wow! π€―ππππ
Thank you @endpts.com for sharing the story about Grace Science, so close to bringing gene therapy to patients with NGly1 disease, hoping for some regulatory flexibility.
endpoints.news/grace-scienc...
ππAlways have a shell in your pocket & sand in your shoes!ππ
#FluorescenceFriday
Multiple #Stapelia having a great time blooming in our conservatory rn. Very hairy, very big, very unique, and very uniquely stinky too! #plantjoy #flypollination #iamabotanist
Society for Developmental Biology
Grace Science CEO Matt Wilsey says FDA demands could kill its gene therapy for the rare and fatal NGLY1 deficiency by September without new funding or a regulatory break.
