Tag Archives: ataluren

Drug company donates £1,000 to ANUK

A global biopharmaceutical company has provided a donation to ANUK for which we are extremely grateful. PTC Therapeutics is the company that discovered and is developing ataluren for the treatment of disorders due to a genetic nonsense mutation. Approximately 20% of patients … Continue reading

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Posted in ANUK news, Fundraising, Research | Tagged , , , | Leave a comment

Cure for aniridia under spotlight

A new study has suggested potential eyedrop therapy for aniridia. Is the hype justified and what are the details? Aniridia is a genetic abnormality caused by mutations in the so-called ‘Master Regulator’ gene for eye development, PAX6.  Because PAX6 is … Continue reading

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Posted in Medical staff talking, Research | Tagged , , , | 7 Comments