Fabry Disease News

April 9, 2021April 9, 2021

‘Break a Sweat for Fabry’ and Other Activities Mark Awareness Month

Fabry Disease News

Activities ranging from sharing educational posters to “breaking a sweat” are underway to mark Fabry Disease Awareness Month, set aside each April to call attention to this rare genetic disorder. ... Read more

April 2, 2021April 2, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

Fabry Disease News

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of ... Read more

March 26, 2021March 26, 2021

Fabrazyme’s Long-term Benefits to Kidneys, Other Organs Added to US Label

Fabry Disease News

Long-term treatment with Fabrazyme (agalsidase beta), an enzyme replacement therapy for people with Fabry disease, slows the progression of kidney disease and significantly delays arriving at outcomes that include heart or kidney ... Read more

March 19, 2021March 19, 2021

NanoGLA, Possibly More Effective ERT for Fabry, Named Orphan Drug in EU

Fabry Disease News

NanoGLA — a potentially more effective version of enzyme replacement therapy that delivers the missing enzyme in Fabry disease through tiny fatty vesicles — has been designated an orphan drug by the European Commission. ... Read more

March 12, 2021March 12, 2021

PRX-102 Safe and Effective Once Monthly Fabry Treatment, Phase 3 Data Show

Fabry Disease News

A once-a-month regimen of the investigational enzyme replacement therapy (ERT) PRX-102 (pegunigalsidase alfa) is safe and effective at maintaining disease stability in adults with Fabry disease, top-line data from the ... Read more

March 8, 2021March 8, 2021

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

COVID-19 News, Fabry Disease News

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, ... Read more

March 2, 2021March 2, 2021

New Test Can Detect Antibodies That Limit ERT, Gene Therapy

Fabry Disease News

Freeline Therapeutics has developed a more robust test to detect the presence of neutralizing antibodies that affect the effectiveness of enzyme replacement therapy (ERT) or gene therapy in people with ... Read more

February 26, 2021February 26, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

COVID-19 News, Fabry Disease News

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 a young age, he also learned ... Read more

February 23, 2021February 23, 2021

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

COVID-19 News, Fabry Disease News

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, research updates, TED-style talks, and a ... Read more

February 23, 2021February 23, 2021

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

Fabry Disease News

One year of treatment with PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy (ERT), safely and effectively slowed kidney disease progression in adults with Fabry disease who were previously treated ... Read more

February 16, 2021February 16, 2021

Ultrasound for Renal Cysts May Hasten Diagnosis

Fabry Disease News

Cysts in the kidneys are more common in people with Fabry disease than in other diseases that affect the kidneys, which is why a new study suggests that looking for ... Read more

February 16, 2021February 16, 2021

Eurodis Survey: Healthcare Experience Worse for Rare Disease Patients

Fabry Disease News

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 16, 2021February 17, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Fabry Disease News

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 10, 2021February 10, 2021

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

COVID-19 News, Fabry Disease News

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the ... Read more

February 9, 2021February 9, 2021

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

Fabry Disease News

The first patient dosed with AVR-RD-01, an investigational gene therapy being tested in the ongoing Phase 2 FAB-GT clinical trial in people with Fabry disease, showed a 100% reduction, or complete ... Read more