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Treatment for CF
- Question
- Is there a treatment/cure for a person with the mutations DF508 & 621+1G>T?
- Answer
- Dear friend,
The treatment of CF, until recently, was symptomatic, i.e. the symptoms are treated and the progress of the pulmonary disease is slowed down with daily physiotherapy and antibiotics, while pancreatic enzymes are administered for pancreatic insufficiency and malabsorption.
Now, since last year, there exists a radical treatment for certain CF mutations. In our CF centre at Agia Sofia Hospital a radical treatment is administered to 5 CF patients (2 with the G551D mutation and three with the G1349D).
For heterozygote and homozygote of DF508 CF mutation (a catetogy, in which you belong), there is underway an experimental treatment combining “661” and Kalydeco. The results are expected by the end of 2016. If they are successful, the treatmend will be available in 2017. For homozygote DF508 patients the substance Orkambi was recently approved for administration in the USA.
Yours friendly,
Dr. Stavros Doudounakis
- 14.08.2015
