Fabry Disease News

January 12, 2021January 12, 2021

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

Fabry Disease News

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 of kidney disease in men and ... Read more

January 5, 2021January 5, 2021

Kidney Function Test Alone May Not Effectively Screen for Fabry

Fabry Disease News

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 to effectively screen for undiagnosed Fabry ... Read more

December 22, 2020December 22, 2020

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

Fabry Disease News

The U.S. Food and Drug Administration (FDA) has extended the review date by three months — to spring 2021 — for a biologics license application (BLA) seeking accelerated approval of ... Read more

December 8, 2020December 9, 2020

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

Fabry Disease News

Using a new harmless version of an adeno-associated virus (AAV), scientists successfully delivered a human version of alpha-galactosidase A — the faulty enzyme in people with Fabry disease — to ... Read more

December 1, 2020December 1, 2020

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

Fabry Disease News

More than half of male patients with classic Fabry disease develop neutralizing antibodies against enzyme replacement therapy (ERT), affecting its effectiveness, according to a study from Japan. This development appeared to be associated ... Read more

November 26, 2020December 28, 2020

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

COVID-19 News, Fabry Disease News

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 health and well-being, according to a ... Read more

November 24, 2020November 24, 2020

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

COVID-19 News, Fabry Disease News

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 Phase 1 and 2 clinical trials, ... Read more

November 17, 2020November 17, 2020

Screen Kidney Transplant Patients for Fabry, Study Recommends

Fabry Disease News

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 the screening was extended to related ... Read more

November 10, 2020November 10, 2020

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

Fabry Disease News

Physicians should consider Fabry disease when trying to determine the cause of recurrent episodes of fever in a patient that have no known or readily identifiable source, a case report highlighted. The ... Read more

November 3, 2020November 3, 2020

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

COVID-19 News, Fabry Disease News

The European Commission has granted orphan drug designation to Avrobio’s investigational gene therapy AVR-RD-01 for the treatment of Fabry disease. The decision, based on a positive opinion from the Committee for ... Read more

October 28, 2020November 30, 2020

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

Fabry Disease News

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in ... Read more

October 27, 2020October 27, 2020

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

Fabry Disease News

Chiesi Global Rare Diseases has launched a campaign called “Rethink Fabry” that seeks to help people with the rare genetic disorder, along with their caregivers and family members, to make ... Read more

October 20, 2020October 20, 2020

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

Fabry Disease News

Protalix BioTherapeutics and Chiesi Global Rare Diseases have launched an expanded access program (EAP) in the U.S. for PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy (ERT) to treat adults ... Read more

October 13, 2020October 13, 2020

Kidney Foundation Opens Effort to Raise Awareness of Fabry Disease

Fabry Disease News

The American Kidney Fund (AKF) has opened a Fabry disease education and awareness campaign that encourages people with chronic kidney disease (CKD) who don’t know the underlying cause of their disorder ... Read more

October 6, 2020November 6, 2020

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

COVID-19 News, Fabry Disease News

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, ... Read more