Article
Patient reported quality of life and medication adherence in Fabry disease patients treated with migalastat: A prospective, multicenter study.
Molecular genetics and metabolism - 1 Feb 2023
Müntze Jonas, Lau Kolja, Cybulla Markus, Brand Eva, Cairns Tereza, Lorenz Lora, Üçeyler Nurcan, Sommer Claudia, Wanner Christoph, Nordbeck Peter
Abstract excerpt
AIMS: Chaperone therapy with migalastat is a novel therapy option in Fabry disease (FD). In contrast to biweekly intravenous enzyme-replacement-therapy in a healthcare setting, oral delivery of migalastat every other day relies on the patient self-administration. Therapy adherence to migalastat and patient reported outcomes have not yet been studied in a real-world scenario. METHODS AND RESULTS: Prospective...
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