This Is What I’m Grateful for Following My Family’s Fabry Diagnoses
Being diagnosed with Fabry disease turned my world upside down. Finding ways to cope and accept my new life, as well as that of ... Read more
Updates
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Rare Disease Film Festival Highlights Patient and Researcher Unity
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 ... Read more -
Managing the Gastrointestinal Symptoms of Fabry Disease
Fabry disease is a rare X-linked genetic disease characterized by the buildup of a type of fat called globotriaosylceramide (Gb3 or GL-3) inside cells, ... Read more -
Kidney Biopsy May Help Diagnose Female Fabry Patients, Case Report Suggests
Kidney biopsies can be instrumental in diagnosing Fabry disease, particularly in females for whom symptoms can vary widely, a new case report contends. The ... Read more -
Walking a Mile in My Mother’s Shoes
They say you never truly know what someone else is going through until you walk a mile in their shoes. Let’s rewind to when ... Read more
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Dealing With Fabry Crises
Fabry disease is a rare genetic disease characterized by the buildup of a type ... -
Rapidly Aging Cells May Contribute to Shorter Lifespan of Fabry Patients, Study Finds
Certain cells in patients with Fabry disease seem to age more rapidly than in ... -
PRX-102 Improves Kidney Function in Fabry Patients, Preliminary Phase 3 Data Show
Treatment with PRX-102 (pegunigalsidase alfa) can safely and effectively improve kidney function in Fabry ... -
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Is Fabry Disease Going to Affect My Life Expectancy?
Fabry disease is a rare genetic disorder characterized by the body’s inability to break ... -
‘Significant’ Levels of Pain Reported by Fabry Patients in Large Survey
Even with treatment, people with Fabry disease often experience pain to an extent that ...
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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