From: Do clinical guidelines facilitate or impede drivers of treatment in Fabry disease?
Drivers | |
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1. Male sex, young age, and clinical findings, such as severe pain and signs/symptoms of organ involvement, are key drivers of early initiation of treatment (4.8) | |
2. Improving clinical outcomes and preventing disease progression are key drivers of early initiation of FD-specific treatment (4.6) | |
3. A family history of FD, especially if severe or with major organ involvement or premature death, is a key driver of early initiation of treatment (4.4) |
Barriers | |
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1. A lack of biomarkers predicting which patients will progress and which will respond to treatment is a key barrier to early initiation of treatment (4.1) | |
2. Misdiagnosis is a key barrier to early initiation of treatment (3.9) |