Please note: While some information will still be current in a year, other information may already be out of date in three months time. If you are in any doubt, please feel free to ask.

cistic fibroses

i would like to know more about the availabilty of Kalydeco in Bulgaria and if there will be any point for someone suffering from CF with mutation 621+1G/T,N1303K to try this medication.and also if not suitable what other treatment should we look for in order to keep quality of life better?


Dear Galina, thank you for your question.

I cannot answer you regarding the specific availability or provision of Kalydeco in Bulgaria. Kalydeco is a relatively new drug and more research needs to be done before it is available for the wider CF patient group. At present Kalydeco is only licenced for the treatment of patients with C.F who have genotype G551D. Further research is currently being carried out to show the effectiveness of the treatment in patients with genotype R117H. Also new studies are being introduced to assess effectiveness of Kalydeco in combination with another drug Lumnicafor, initially these studies are focusing on patients with genotypeF 508del; F508del.
To ensure that you maintain your optimum health I think it is important to ensure you attend hospital appointments and have regular checkups with your CF specialist who will assess your personal needs and develop a specific plan tailored to your specific health care needs.
It is important to take time to work out and get into a good routine of carrying out your treatments, be regimented and committed to execute this daily.
Exercise is important to maintaining and improving your health, if you can find an exercise that you find enjoyable you are more likely to make this a a part of your daily activities. If you don’t have this in place already spend a little time working out the amount of time you can spend, and what is realistic in terms of cost and convenience for you, set yourself achievable goals and targets and enjoy working towards meeting them.
Engage with the dietician in your clinic and ask her/him to provide you with some advice and help to ensure your nutritional needs are being met and to help develop the best diet plan tailored for your specific likes and dislikes.
I hope you find this information useful, best wishes for your future.

Kind regards,

Vivienne McClenaghan
Prof Stuart Elborn
The answer is edited by: Prof Stuart Elborn