Updates

January 22, 2020January 22, 2020
No Race Component In Galafold Effectiveness, Subgroup Analysis Suggests

Galafold (migalastat) is an effective treatment for certain Japanese people with Fabry disease, with no apparent differences in response to treatment due to race, ... Read more
January 16, 2020January 17, 2020
Enzyme Replacement Therapy for Fabry Disease — What to Keep in Mind

Enzyme replacement therapy (ERT), such as Fabrazyme, addresses the primary cause of Fabry disease — mutations in the GLA gene that lead to the ... Read more
January 15, 2020January 15, 2020
Imaging Technique That Detects Changes in Eye Blood Vessels May Help in Early Diagnosis of Fabry, Study Says

Detectable differences in blood vessel architecture in the eyes of people with Fabry disease could prove useful in diagnosing the disease early, a study ... Read more
January 9, 2020January 10, 2020
Cardiac Care in Fabry Disease

Heart disease is common in Fabry disease patients, and those with the disease should be vigilant about taking steps to optimize their cardiac care. ... Read more
January 8, 2020January 8, 2020
High-risk Screenings for Fabry Among Chronic Kidney Disease Patients Should Use Biomarkers Other Than Gb3, Study Says

High-risk screenings for Fabry disease among individuals with chronic kidney disease (CKD) should be based on biomarkers other than globotriaosylceramide (Gb3) to lower the ... Read more

Columns, The Fabry Files - a Column by Eunice White

How I Found My Road to Exercise
January 8, 2020January 7, 2020
It’s 4:45 a.m. and the alarm is sounding. What comes to mind when you ...
News

Cardiac Health Checks Should Be Given Women with GLA Mutations, Finish Study Suggests
December 18, 2019December 18, 2019
A common GLA mutation is highly associated with the development of Fabry disease in ...
Chronically Caring – a Column by Morgan Yontz, Columns

Facing Fears About Fabry After a Hospital Admission
December 17, 2019December 13, 2019
In recent weeks I have had a traumatic medical experience that has affected me ...
News

Gene Therapy Takes Center Stage at 2019 NORD Summit
December 11, 2019December 10, 2019
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic ...
News

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
December 6, 2019December 6, 2019
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or ...
News

Investigators Discover New GLA Mutation Likely Associated With Fabry Disease, Case Report Shows
December 4, 2019December 4, 2019
Korean investigators have discovered a new mutation in the GLA gene — located on ...

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Kidney Care in Fabry Disease
December 4, 2019December 6, 2019
Kidney problems are common in people with Fabry disease. They are caused by the deposition ...
Answering the Hard Question for Which No Parent Is Prepared
December 4, 2019December 2, 2019
“Mom, can Fabry kill you?” This wasn’t the first time this question was posed ...
Dealing With Sweating Abnormalities When You Have Fabry Disease
November 28, 2019November 28, 2019
Protalix and Chiesi Get FDA OK to Seek Accelerated Approval of PRX-102 for Fabry Disease
November 27, 2019December 13, 2019
GLA Gene Variant Found to Trigger Kidney Disease Associated With Fabry, Family Study Shows
November 20, 2019November 20, 2019
This Is What I’m Grateful for Following My Family’s Fabry Diagnoses
November 19, 2019November 15, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity
November 13, 2019
Rare disease-themed videos glowed on a large screen before an audience of people in ...
Managing the Gastrointestinal Symptoms of Fabry Disease
November 8, 2019
Kidney Biopsy May Help Diagnose Female Fabry Patients, Case Report Suggests
November 6, 2019November 6, 2019
Walking a Mile in My Mother’s Shoes
November 6, 2019November 4, 2019

Dealing With Fabry Crises
November 1, 2019
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
October 30, 2019October 30, 2019
PRX-102 Improves Kidney Function in Fabry Patients, Preliminary Phase 3 Data Show
October 23, 2019October 23, 2019

Chronically Caring – a Column by Morgan Yontz, Columns

Learning to Make My Voice Heard
October 22, 2019October 18, 2019
I have always been the type of person to go out of my way ...
Health Insights

Is Fabry Disease Going to Affect My Life Expectancy?
October 21, 2019
Fabry disease is a rare genetic disorder characterized by the body’s inability to break ...
News

‘Significant’ Levels of Pain Reported by Fabry Patients in Large Survey
October 16, 2019October 16, 2019
Even with treatment, people with Fabry disease often experience pain to an extent that ...
Columns, The Fabry Files - a Column by Eunice White

Attending a Fantastic Family Camp Full of ‘Fabbers’
October 16, 2019November 1, 2019
Fabber. Have you ever heard that term? It’s used to describe those with Fabry ...
Health Insights

What to do After You’re Diagnosed with Fabry Disease
October 11, 2019October 11, 2019
When you’re diagnosed with Fabry disease, it’s common to be surprised and frightened about ...
News

Enrollment Completed in Phase 3 BALANCE Trial of PRX-102 for the Treatment of Fabry Disease
October 9, 2019October 9, 2019
Enrollment has been completed for a Phase 3 clinical trial comparing PRX-102 (pegunigalsidase alfa) ...

February 6, 2018February 6, 2018

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

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