Updates

September 28, 2020September 28, 2020
Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses ... Read more
September 25, 2020September 25, 2020
Reproductive Options for People With Fabry Disease

The thought of potentially passing Fabry disease on to your children can be daunting. However, there are options to limit this risk and help ... Read more
September 23, 2020August 22, 2020
My Childhood Made Sense After My Diagnosis

I had a happy childhood. It was fun to grow up in a large family with six brothers and one sister, especially during the ... Read more
September 17, 2020September 18, 2020
Prenatal Genetic Diagnosis for Fabry Disease

Prenatal genetic diagnosis can be a useful tool to detect genetic disorders such as Fabry disease. The following information should help you understand the ... Read more
September 15, 2020September 15, 2020
Long-Term Galafold Safe, Effective as Fabry Treatment, ATTRACT Extension Shows

The results from a one-year extension study of the Phase 3 ATTRACT trial demonstrate the long-term safety and effectiveness of Galafold (migalastat) in people with ... Read more

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

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

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

Reproductive Options for People With Fabry Disease

My Childhood Made Sense After My Diagnosis

Prenatal Genetic Diagnosis for Fabry Disease

Long-Term Galafold Safe, Effective as Fabry Treatment, ATTRACT Extension Shows

Newborn Screening for Fabry Disease

Activities Underway for Newborn Screening Awareness Month

ST-920 Gene Therapy Found Safe, Effective in Mouse Model; Supports First-In-Human Trial

FDA Reviewing Application of PRX-102 as Fabry Treatment

Same But Different Photo Contest Celebrates People With Rare Diseases

Raising Awareness of Fabry Disease

Subscribe to our mailing list

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

FDA Puts Fabry Gene Therapy 4D-310 on Fast Track

What Do Blood Test Results Mean in Fabry Disease?

Managing Fabry Disease Requires a Balancing Act

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Mindfulness for Fabry Disease Patients

Pain, Negative Health Perceptions Linked to Patient Depression, Study Finds

New Streaming Channel Showcases Rare Disease Films

Creating a Treatment Plan for Fabry Disease

My Daughter Is My Hero

New Mutation That Causes Fabry Found in GLA Gene, Case Study Reports

FAQs About a Kidney Transplant for Fabry Disease

Fabry Disease: A Road Less Traveled

Certain Fabry Patients May Switch from ERT to Galafold, Real-life Study Suggests

A Look at Fabry Through My Daughter’s Eyes

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

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

Pin It on Pinterest

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

Reproductive Options for People With Fabry Disease

My Childhood Made Sense After My Diagnosis

Prenatal Genetic Diagnosis for Fabry Disease

Long-Term Galafold Safe, Effective as Fabry Treatment, ATTRACT Extension Shows

Newborn Screening for Fabry Disease

Activities Underway for Newborn Screening Awareness Month

ST-920 Gene Therapy Found Safe, Effective in Mouse Model; Supports First-In-Human Trial

FDA Reviewing Application of PRX-102 as Fabry Treatment

Same But Different Photo Contest Celebrates People With Rare Diseases

Raising Awareness of Fabry Disease

Subscribe to our mailing list

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

FDA Puts Fabry Gene Therapy 4D-310 on Fast Track

What Do Blood Test Results Mean in Fabry Disease?

Managing Fabry Disease Requires a Balancing Act

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Mindfulness for Fabry Disease Patients

Pain, Negative Health Perceptions Linked to Patient Depression, Study Finds

New Streaming Channel Showcases Rare Disease Films

Creating a Treatment Plan for Fabry Disease

My Daughter Is My Hero

New Mutation That Causes Fabry Found in GLA Gene, Case Study Reports

FAQs About a Kidney Transplant for Fabry Disease

Fabry Disease: A Road Less Traveled

Certain Fabry Patients May Switch from ERT to Galafold, Real-life Study Suggests

A Look at Fabry Through My Daughter’s Eyes

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

Pin It on Pinterest