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pct drug

hello, i would like to know if the pct drug can be used on haemophiliacs.
I am assuming that the PTC drug you are referring to is the drug that is being examined in a current clinical trial which is entitled- A Phase 3 Efficacy and Safety Study of PCT124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis.” To my knowledge this PTC124 study is due to commence mid 2009 and will take place across 13 countries, and aims to recruit up to 208 subjects.
This study is a cystic fibrosis study and I am not aware of any studies being done
with this drug in haemophiliac populations.

With respect to cystic fibrosis I would like to highlight that if this study is successful, the availability of the drug in clinic may still not be for a few years as the regulatory process for new drugs is very rigorous.

I hope this answer addresses your query


Judy Bradley
The answer is edited by: Prof Stuart Elborn