Fabrazyme is an approved treatment for Fabry disease patients age 8 and older. It was developed by Sanofi Genzyme. Following are answers to some frequently asked questions about the treatment. How does Fabrazyme work? Fabrazyme is an enzyme replacement therapy. In patients with Fabry…
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…
A first patient has been treated with PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy for Fabry disease that is under review for approval, through an expanded access program (EAP) in the United States. “The initiation of treatment in the first patient enrolled in our EAP is an important milestone for the Fabry…
Hearing loss and vertigo are common symptoms of Fabry disease. This article discusses how Fabry disease may affect your hearing, and looks at ways that doctors may diagnose and treat hearing problems. How do hearing and balance work? Hearing is a complicated process that involves multiple structures in…
PRX-102 (pegunigalsidase alfa), an experimental enzyme replacement therapy (ERT) for Fabry disease, safely led to significant improvements in kidney function and slowed the progression of kidney disease in men and women previously treated with Replagal (agalsidase alfa). These are the final results from the open-label Phase 3 BRIDGE…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
Fabry disease leads to heart complications in more than 50% of patients. Here is more information about these complications and how doctors diagnose and treat them. Heart problems in Fabry disease Patients with Fabry disease may experience a number of cardiac problems. The most frequent is left ventricular hypertrophy,…
Using only an age-dependent threshold of estimated glomerular filtration rate (eGFR) — a marker of kidney function — indicating mild-to-moderate kidney disease is insufficient to effectively screen for undiagnosed Fabry disease patients among the general population, a study shows. The findings suggest that appropriate screening likely wil require evidence…
The U.S. Food and Drug Administration (FDA) has extended the review date by three months — to spring 2021 — for a biologics license application (BLA) seeking accelerated approval of PRX-102 (pegunigalsidase alfa) to treat adults with Fabry disease. Protalix BioTherapeutics and Chiesi Global Rare Diseases, who are…
I am a 66-year-old male with Fabry disease, which causes many adverse and potentially life-threatening symptoms. As I look back on some of my major life events, I often think about the saying, “A cat has nine lives.” I’ve had many encounters due to both Fabry disease complications and…
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