Updates

November 26, 2020November 26, 2020
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their ... Read more
November 24, 2020November 24, 2020
Gene Therapy Showing Efficacy Years After Use in Clinical Trials, Avrobio Says

With data now reaching up to 3.5 years, benefits are continuing to be reported with the use of AVR-RD-01, Avrobio‘s experimental gene therapy for Fabry disease, in ... Read more
November 19, 2020November 17, 2020
Having Fabry While in the Army Was Like Mixing Oil and Water

As a boy, I wasn’t aware Fabry disease caused the many physical issues I experienced, such as a reduced ability to perspire (called hypohidrosis), ... Read more
November 17, 2020November 17, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and ... Read more
November 17, 2020November 17, 2020
Screen Kidney Transplant Patients for Fabry, Study Recommends

A genetic screening study found a higher-than-expected prevalence of undiagnosed Fabry disease in people who had kidney failure and transplant without a known cause. When ... 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

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

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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

Fabrazyme vs. Replagal for the Treatment of Fabry Disease

Kidney Foundation Opens Effort to Raise Awareness of Fabry Disease

Independent Living for Fabry Disease Patients

The Challenges of Parenting With Fabry Disease

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

Preimplantation Genetic Diagnosis (PGD) for Fabry Disease

Using Telemedicine Ensures Patient Monitoring and Safety in Emergencies, Study Says

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

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