Ahmet AKICI, Volkan AYDIN, Narin AKICI, N. İpek KIRMIZI, Banu BAYAR

Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey

Background/aim: Pediatric patients, especially those with rare diseases, represent a population that has a high tendency towards offlabel drug use (OLDU) and needs a more careful practice of pharmacotherapy than in adults. We aimed to investigate biotechnological drug use in children with rare diseases requiring OLDU. Materials and methods: This retrospective study examined all single-diagnosed OLDU applications (n= 5792) for 4992 children (

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