Updates

March 5, 2021March 5, 2021
FAQs About Replagal

Replagal is a treatment for Fabry disease patients ages 7 and older. Shire (now owned by Takeda) developed it. Although in use in many ... Read more
March 2, 2021March 2, 2021
New Test Can Detect Antibodies That Limit ERT, Gene Therapy

Freeline Therapeutics has developed a more robust test to detect the presence of neutralizing antibodies that affect the effectiveness of enzyme replacement therapy (ERT) ... Read more
February 26, 2021February 26, 2021
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from ... Read more
February 26, 2021February 26, 2021
Building Emotional Resilience When You Have Fabry Disease

Emotional resilience is the ability to adapt to stressful situations and cope with life’s ups and downs. Here’s how you can develop emotional resilience ... Read more
February 23, 2021February 23, 2021
Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, ... 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

FAQs About Replagal

New Test Can Detect Antibodies That Limit ERT, Gene Therapy

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

PRX-102 Slows Kidney Disease Progression in Fabry Patients Previously on Replagal

Fabry Disease and Shoshin

In Managing Fabry Symptoms, We Must Leave No Stone Unturned

Ultrasound for Renal Cysts May Hasten Diagnosis

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Importance of Advocacy for Fabry Disease

Subscribe to our mailing list

Pandemic Won’t Stop Rare Disease Day on Feb. 28

AVR-RD-01 Gene Therapy Clears Fabry Patient of Toxic Substrate, Trial Reports

Respite Care for Fabry Disease

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

I Have Fabry Disease. How Long Will I Live?

Charitable Program by Takeda Helps Patients Worldwide Gain Treatment

Preparing for ERT Infusion for Fabry Disease

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Online Tool Could Help Users Find Possible Causes of Their Symptoms

FAQs About Fabrazyme for Fabry Disease

NORD’s Caregiver Respite Program Continues Through Pandemic

PRX-102 Given to 1st Patient in US Under Expanded Access Program

Fabry Disease and Hearing

PRX-102 Shown to Safely Improve Kidney Function in Adults With Fabry

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Fabry Disease and the Heart

Kidney Function Test Alone May Not Effectively Screen for Fabry

FDA Extends Review Date to April for US Approval of PRX-102

I Feel Like a Cat With 9 Lives

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

FAQs About Replagal

New Test Can Detect Antibodies That Limit ERT, Gene Therapy

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Building Emotional Resilience When You Have Fabry Disease

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

PRX-102 Slows Kidney Disease Progression in Fabry Patients Previously on Replagal

Fabry Disease and Shoshin

In Managing Fabry Symptoms, We Must Leave No Stone Unturned

Ultrasound for Renal Cysts May Hasten Diagnosis

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Importance of Advocacy for Fabry Disease

Subscribe to our mailing list

Pandemic Won’t Stop Rare Disease Day on Feb. 28

AVR-RD-01 Gene Therapy Clears Fabry Patient of Toxic Substrate, Trial Reports

Respite Care for Fabry Disease

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

I Have Fabry Disease. How Long Will I Live?

Charitable Program by Takeda Helps Patients Worldwide Gain Treatment

Preparing for ERT Infusion for Fabry Disease

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Online Tool Could Help Users Find Possible Causes of Their Symptoms

FAQs About Fabrazyme for Fabry Disease

NORD’s Caregiver Respite Program Continues Through Pandemic

PRX-102 Given to 1st Patient in US Under Expanded Access Program

Fabry Disease and Hearing

PRX-102 Shown to Safely Improve Kidney Function in Adults With Fabry

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Fabry Disease and the Heart

Kidney Function Test Alone May Not Effectively Screen for Fabry

FDA Extends Review Date to April for US Approval of PRX-102

I Feel Like a Cat With 9 Lives