Updates

July 14, 2020July 14, 2020
Alternative Therapies Needed for Patients Who Respond Poorly to ERT, Case Report Says

A case study of two cousins with Fabry disease who started producing high levels of neutralizing antibodies after enzyme replacement therapy (ERT) highlight the ... Read more
July 14, 2020July 14, 2020
The Fabry Registry

If you have Fabry disease, you should consider joining the Fabry Registry. Among other benefits, the information you can provide may help scientists develop ... Read more
July 9, 2020July 9, 2020
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, ... Read more
July 7, 2020July 7, 2020
Comprehensive New Urine Test May Help Doctors Monitor Fabry Disease Progression

A newly developed urine test that measures the levels of different proteins may help doctors to more quickly and accurately monitor the progression of ... Read more
June 30, 2020June 30, 2020
Single Dose of AMT-190 Effective in Mice and Primate Studies of Fabry

A single administration of AMT-190, uniQure’s investigational gene therapy for Fabry disease, significantly increases the activity of alpha-galactosidase A — the faulty enzyme in ... Read more

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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

Updates

Alternative Therapies Needed for Patients Who Respond Poorly to ERT, Case Report Says

The Fabry Registry

Comprehensive New Urine Test May Help Doctors Monitor Fabry Disease Progression

Single Dose of AMT-190 Effective in Mice and Primate Studies of Fabry

Women With Fabry Disease

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

FAQs About Kidney Health and Dialysis in Fabry Disease

Clinical Trial Data Support AVR-RD-01 Gene Therapy for Fabry Disease

Fabry Disease and Your Eyes

Coalition Will Address Racial Disparities in Rare Disease Communities

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How to Beat the Summer Heat

PRX-102 ERT Improves Kidney Function in Patients Treated With Replagal, Trial Shows

Clinical Trials for Fabry Disease

Protalix, Chiesi Request U.S. Approval of PRX-102 to Treat Fabry in Adults

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Genetic Testing for Fabry Disease: What to Expect?

Fewer Antibodies Against PRX-102 Than Fabrazyme Likely, Trial Data Suggest

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

The Spoon Theory and Fabry Disease

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Genetic Counseling for Fabry Disease

FDA Gives Fabry Gene Therapy, FLT190, Orphan Drug Status

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Vitamin D Deficiency and Fabry Disease

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Protalix’s Pegunigalsidase Alfa Granted FDA Fast Track Status for Fabry Disease

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Alternative Therapies Needed for Patients Who Respond Poorly to ERT, Case Report Says

The Fabry Registry

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

Comprehensive New Urine Test May Help Doctors Monitor Fabry Disease Progression

Single Dose of AMT-190 Effective in Mice and Primate Studies of Fabry

Women With Fabry Disease

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

FAQs About Kidney Health and Dialysis in Fabry Disease

Clinical Trial Data Support AVR-RD-01 Gene Therapy for Fabry Disease

Fabry Disease and Your Eyes

Coalition Will Address Racial Disparities in Rare Disease Communities

Subscribe to our mailing list

How to Beat the Summer Heat

PRX-102 ERT Improves Kidney Function in Patients Treated With Replagal, Trial Shows

Clinical Trials for Fabry Disease

Protalix, Chiesi Request U.S. Approval of PRX-102 to Treat Fabry in Adults

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Genetic Testing for Fabry Disease: What to Expect?

Fewer Antibodies Against PRX-102 Than Fabrazyme Likely, Trial Data Suggest

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

The Spoon Theory and Fabry Disease

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Genetic Counseling for Fabry Disease

FDA Gives Fabry Gene Therapy, FLT190, Orphan Drug Status

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Vitamin D Deficiency and Fabry Disease

I Always Have Something to Be Thankful For

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

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