Making the Most of Your Doctor’s Visit When You Have Fabry Disease
Fabry disease is a rare genetic disorder, which can make doctor visits much more complicated. Here are some tips to ensure that your doctor’s ... Read more
Updates
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Genetics of Fabry Disease
Like all genetic diseases, Fabry disease often poses a major concern for parents and caregivers of an affected child. Understanding the genetics of the ... Read more -
New GLA Mutation Leading to Low Enzyme Activity and Kidney Disease Found in Chinese Patient
A new mutation in the GLA gene was found in a Chinese patient with Fabry disease that leads to low alpha-galactosidase A (alpha-GAL A) ... Read more -
FAQs in Fabry Disease
Patients and caregivers of genetic disorders such as Fabry disease often have more questions than answers due to the paucity of easily accessible information. ... Read more
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At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is ... -
Enzyme Testing for Fabry Disease May Be More Cost-Effective Than Genetic Tests, Study Says
Testing alpha-galactosidase A enzyme activity may be a more cost-effective screening method for diagnosing ... -
Amicus CEO John Crowley Outlines Progress in Fabry, Pompe, Batten Therapies
Pharmaceutical executives rarely make for a sympathetic Hollywood medical drama. But John Crowley did, ... -
The Start of the Journey
Welcome to Morgan Yontz’s “Chronically Caring,” a new Fabry Disease News column. The first step ... -
Dried Blood Drops May Help Screen At-risk Patients Early for Fabry Disease, Study Suggests
Analysis of dry blood drops may help diagnose Fabry disease and begin early treatment ...
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Protalix’s Pegunigalsidase Alfa Granted FDA Fast Track Status for Fabry Disease
News
The U.S. Food and Drug Administration (FDA) has granted fast track designation to pegunigalsidase alfa, or PRX-102, Protalix BioTherapeutics’ therapy candidate under development for Fabry disease. FDA’s fast track status is designed to facilitate the development and speed up FDA review ... Read more