Updates

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
June 26, 2020June 26, 2020
Women With Fabry Disease

Fabry disease is a rare genetic disorder that is caused by mutations in the GLA gene. The body uses the instructions that this gene provides to ... Read more
June 23, 2020June 23, 2020
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization ... Read more
June 19, 2020June 19, 2020
FAQs About Kidney Health and Dialysis in Fabry Disease

Fabry disease is a rare disease characterized by the buildup of a fatty molecule (globotriaosylceramide Gb3 or GL-3) inside cells. This accumulation can lead to ... 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

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

How to Beat the Summer Heat

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

Clinical Trials for Fabry Disease

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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

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

A143T Mutation in GLA Gene Likely Causes Late-onset Fabry Disease, Study Suggests

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

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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

How to Beat the Summer Heat

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

Clinical Trials for Fabry Disease

Subscribe to our mailing list

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

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

A143T Mutation in GLA Gene Likely Causes Late-onset Fabry Disease, Study Suggests

Pin It on Pinterest