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Article

Preventive study in subjects at risk of fatal familial insomnia: Innovative approach to rare diseases

4 Mar 2015

Abstract excerpt

The text describes a preventive clinical trial with drug treatment in a very rare neurodegenerative disease (Fatal familial Insomnia, FFI) designed with the help of individuals at genetic risk of developing the disease, asymptomatic carriers, who have agreed to be exposed over a 10-year period to doxycycline, an antibiotic with anti-prion activity. At least 10 carriers of the FFI mutation over 42 y old will be...

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PMID: 25996399
DOI: 10.1080/19336896.2015.1027857

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