Long-Term Effects of Enzyme Replacement Therapy for Anderson-Fabry Disease.

We report the differences in the clinical effects of ERT continued for > 10 years in three patients with the same genotype. Left ventricular hypertrophy and myocardial dysfunction progressed in the heterozygote proband even under ERT, although disease progression was prevented in two sons of Case 1.
PMID: 30464119 [PubMed - as supplied by publisher]

Source: International Heart Journal - November 24, 2018 Category: Cardiology Tags: Int Heart J Source Type: research