Let's make rare disease treatments less rare

ABPI President Haseeb Ahmad says that genome sequencing and advanced therapies give hope to people with rare diseases and their families – we have to get them into the hands of patients.

‘Everything is theoretically impossible, until it is done’ – that’s a quote from a famous science fiction author, but science fact is proving him to be increasingly right again and again.

Harnessing the power of genome sequencing and genetic therapies, science is now coming up with new medicines and treatments – and potentially cures – for rare diseases that were previously thought impossible to treat.  

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  • Rare diseases