#curecdkl5 My son talking about his sister - follow the link to read his story near the end titled “Inspirational Amber”

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.

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 👇


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...

#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!

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