Last Updated: 09/26/2026
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Found 4 publications
Global reach of over 20 years of experience in the patient-centered Fabry Registry: Advancement of Fabry disease expertise and dissemination of real-world evidence to the Fabry community.
Journal: Molecular genetics and metabolism
Published: February 17, 2023
Pregnancy Outcomes in Late Onset Pompe Disease.
Journal: Life (Basel, Switzerland)
Published: August 25, 2020
Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.
Journal: The New England journal of medicine
Published: August 11, 2016
Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: May 06, 2015
Showing 1-4 of 4
Last Updated: 09/26/2026