‘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 it affects their quality of life, a study based on ... Read more
Updates
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Attending a Fantastic Family Camp Full of ‘Fabbers’
Fabber. Have you ever heard that term? It’s used to describe those with Fabry disease. I recently spent a weekend with my family and ... Read more -
What to do After You’re Diagnosed with Fabry Disease
When you’re diagnosed with Fabry disease, it’s common to be surprised and frightened about what this means for your quality of life and your ... Read more -
Enrollment Completed in Phase 3 BALANCE Trial of PRX-102 for the Treatment of Fabry Disease
Enrollment has been completed for a Phase 3 clinical trial comparing PRX-102 (pegunigalsidase alfa) with Fabrazyme (agalsidase beta) for the treatment of Fabry disease. The ... Read more -
Tips for Managing Stress When You Have Fabry Disease
Fabry disease is a rare genetic disorder that impairs the body’s ability to break down a type of fat called globotriaosylceramide (Gb3 or GL-3), ... Read more
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Fabry Disease Patients with Higher Residual GLA Activity Respond Better to Galafold Treatment, Swiss Study Says
Nearly half of Fabry patients have amenable or responsive mutations to Galafold (migalastat) but ... -
Tips for Fabry Disease Caregivers
A person with a rare genetic disease, such as Fabry disease, often needs extra ... -
NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, ... -
Heart Cells Grown in Lab Dish May Help in Understanding Fabry’s Cardiac Complications
Heart cells derived from patients’ stem cells and grown in a lab dish can ... -
Transitioning to Home Infusions: Expectation vs. Reality
I was amazed by all of the support available to us at the beginning ... -
First Patient Dosed in Phase 1/2 Trial of Fabry Disease Gene Therapy Candidate FLT190
The first patient in a Phase 1/2 clinical trial of Fabry disease gene therapy ...
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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