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#curecdkl5 My son talking about his sister - follow the link to read his story near the end titled “Inspirational Amber” https://t.co/AZlAzRA1BK

Did you know? A #raredisease is defined as one that affects less than 5 in 10,000 of the general population? Visit #RAREfest18 - an interactive, vibrant exhibition, talks and films event. https://t.co/p29jsfg703

We are proud to announce our first step into gene therapy and the expansion of our pipeline, with the potential to transform the lives of 10,000+ children with fatal genetic diseases #BattenDisease #RareCompany

One fo the great uses of #OpenScience: researchers from the Francis Crick Institute have found key substrates fo the #CDKL5 protein that can help us develop new medicines for a very bad #RareDisease and have shared the entire dataset online! Link 👇

@TheCrick @CDKL5USA @CDKL5UK https://t.co/WBD4Fzn3lZ

Il 1 settembre si è tenuta a Marghera la quinta edizione della Corri con Noi, una giornata che regala sempre grandi emozioni a chi vi partecipa. Una corsa, a volte una camminata, ma sempre in compagnia... https://t.co/zNussjntrR

#curecdkl5 #cdkl5champions Only a couple of days and these great people will start their charity climb of the Kilimanjaro!

If you can sponsor them please do so!

https://t.co/jiOj7hkul5 https://t.co/o8fqTb8Cmr

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