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Toxicity spectrum of pegvaliase: A pharmacovigilance analysis using the FAERS database.
Smoking and alcohol, health-related quality of life and psychiatric comorbidities in Leber's Hereditary Optic Neuropathy mutation carriers: a prospective cohort study.
Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients.
Globotriaosylsphingosine (lyso-Gb3) might not be a reliable marker for monitoring the long-term therapeutic outcomes of enzyme replacement therapy for late-onset Fabry patients with the Chinese hotspot mutation (IVS4+919G>A).