Updates

January 15, 2021January 15, 2021
Fabry Disease and Hearing

Hearing loss and vertigo are common symptoms of Fabry disease. This article discusses how Fabry disease may affect your hearing, and looks at ways ... Read more
January 12, 2021January 12, 2021
PRX-102 Shown to Safely Improve Kidney Function in Adults With Fabry

PRX-102 (pegunigalsidase alfa), an experimental enzyme replacement therapy (ERT) for Fabry disease, safely led to significant improvements in kidney function and slowed the progression ... Read more
January 12, 2021January 12, 2021
NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual ... Read more
January 8, 2021January 8, 2021
Fabry Disease and the Heart

Fabry disease leads to heart complications in more than 50% of patients. Here is more information about these complications and how doctors diagnose and ... Read more
January 5, 2021January 5, 2021
Kidney Function Test Alone May Not Effectively Screen for Fabry

Using only an age-dependent threshold of estimated glomerular filtration rate (eGFR) — a marker of kidney function — indicating mild-to-moderate kidney disease is insufficient ... 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

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

EURORDIS Award Contest Welcomes Photos of Life With Rare Disease

Multidisciplinary Care Team for Fabry Disease

Recreational Therapy for Fabry Disease Patients

New Strategy Used in Fabry Mouse Model Holds Potential for Gene Therapy

Subscribe to our mailing list

Sleep Hygiene for Fabry Disease

Study: Most Males With Classic Fabry Develop Neutralizing Antibodies Against ERT

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Gene Therapy Showing Efficacy Years After Use in Clinical Trials, Avrobio Says

Having Fabry While in the Army Was Like Mixing Oil and Water

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Screen Kidney Transplant Patients for Fabry, Study Recommends

Fabry Disease and Sleep

Fabry Possible in People With Recurrent and Unexplained Fevers, Case Report Says

Tips for Managing Fatigue When You Have Fabry Disease

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

AVR-RD-01 Gene Therapy Granted Orphan Drug Status in EU

Fabry Disease and Seizures

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

Chiesi Global Launches ‘Rethink Fabry’ Campaign With New Resources, Website

Fabry Disease and Anesthesia

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

Protalix, Chiesi Launch Expanded Access Program for PRX-102 in the US

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