Effects of Current Therapies on Disease Progression in Fabry Disease: A Narrative Review for Better Patient Management in Clinical Practice.

Although the extent of the non-classical phenotype may be initially less when compared to the classical disease presentation, the severity of individual renal or cardiac manifestations in advanced stages of patients with late-onset variants may be significant enough to require renal replacement therapy or pacemaker/cardioverter defibrillator implantation.

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In a comparative trial, migalastat treatment for 18 months led to a small, but statistically significant, reduction in LVMI; in contrast, the reduction in LVMI with agalsidase alfa or agalsidase beta did not reach statistical significance [ 60 ].

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