A Charity dedicated to helping children WORLDWIDE suffering the disabling ultra-RARE brain disease; Aromatic Amino Acid Decarboxylase (AADCd) Deficiency
AADC is one of the few IMD for which gene therapy has moved from the lab into clinical use. Upstazta was approved for use earlier this year and Roubertier et al discuss the necessity of a framework for a quality-assured application of its use.
https://t.co/TsI5heqfc8
@AADCTRUST
Would’ve enjoyed watching this presentation, especially as a patient organisation who drove this therapy forward and knowing all the benefactors personally! 👏🏻👏🏻👏🏻❤️
The graphics in this article are excellent— this is a huge part of communications that has been missing. If you want to understand how layered interventions reduce #covid19 spread— why masks are indispensable but not always “enough” — read this.
https://t.co/r1KfeKHW5J
What a little CHRISTMAS CRACKER Huzaifah is!
Our gorgeous little boy from the UK, in his Spiderman PJs, has now received AAV2-hAADC Gene Therapy Treatment in Poland. A Christmas gift that I am sure Huzaifah... https://t.co/X5oMggtqIu
On the 10th DAY OF CHRISTMAS, we opened the door of our advent calendar to find a truly special gift was given…
Our handsome boy, 12 year old Marcus from the UK received AAV2-hAADC Gene Therapy in Poland, a... https://t.co/TtgqhbgRPi
@eightth may we ask if you are using this twitter name? we’d really like to use if it was possible. Please can you let me know, thank you 🙏🏻 so much Lisa
We are absolutely THRILLED to share the news with you that Swathi, aged 19, from the USA, has now received AAV2-hAADC Gene Therapy Treatment in Poland. She is the 3rd young ADULT to receive the pioneering brain... https://t.co/zexkpR4G4M
Hallie has an ultra rare brain condition which has left her essentially locked in her own body. They’re hoping the operation may pave the way for her to walk and talk and stop her daily seizures @GranadaReports https://t.co/pYflZTtNtd
On @GranadaReports tonight I’m in Poland following 6 year old Hallie as she becomes only the 8th child to undergo experimental brain surgery. Our viewers helped family raise £70,000 for the operation #aadc#genetherapy#hopeforhallie