Posts by: Alice Melao

News

Treatment with PRX-102 (pegunigalsidase alfa) can safely and effectively improve kidney function in Fabry disease patients, according to preliminary data from a Phase 3 clinical trial. One year of ... Read more

October 9, 2019October 9, 2019

Enrollment Completed in Phase 3 BALANCE Trial of PRX-102 for the Treatment of Fabry Disease

News

Enrollment has been completed for a Phase 3 clinical trial comparing PRX-102 (pegunigalsidase alfa) with Fabrazyme (agalsidase beta) for the treatment of Fabry disease. The BALANCE trial (NCT02795676) estimated enrolling ... Read more

August 14, 2019August 14, 2019

Dried Blood Drops May Help Screen At-risk Patients Early for Fabry Disease, Study Suggests

News

Analysis of dry blood drops may help diagnose Fabry disease and begin early treatment in patients who are experiencing cardiac, renal, or neurological symptoms of unknown cause, according to ... Read more

August 7, 2019August 7, 2019

Galafold Approved to Treat Fabry Teens and Adults in Argentina, Amicus Announces

News

Adolescents and adults with Fabry disease who live in Argentina can now be treated with Amicus Therapeutics’ oral precision medicine Galafold (migalastat). The Agency of Medicines, Food and Medical ... Read more

July 24, 2019July 24, 2019

Gene Therapy AVR-RD-01 Effectively Prevents Buildup of Toxic Gb3 in Fabry Patients, Interim Data Show

News

Avrobio’s investigational gene therapy AVR-RD-01 shows potential in promoting durable beneficial effects to treat Fabry disease, with enhanced therapeutic activity compared with the standard-of-care enzyme replacement therapy, according to ... Read more

June 19, 2019June 19, 2019

Protalix, Chiesi Preparing to Request Accelerated FDA Approval of PRX-102 for Fabry Disease

News

Protalix BioTherapeutics and Chiesi Farmaceutici are planning to submit a biologics license application with the U.S. Food and Drug Administration (FDA) for PRX-102 (pegunigalsidase alfa) as a treatment for ... Read more

February 13, 2019February 13, 2019

Phase 3 Trial Shows Increased Stability of PRX-102 Over Other ERTs for Fabry Disease

News

PRX-102 (pegunigalsidase alfa) is safe for the treatment of patients with Fabry disease and has better stability over other available therapies, according to interim data from a Phase 3 ... Read more

December 18, 2018December 18, 2018

LysoGb3 Blood Levels Can Help Diagnose Fabry Disease, Large Study Shows

News

Globotriaosylsphingosine (LysoGb3) blood levels can be a useful and accurate biomarker to diagnose and monitor Fabry disease, rather than only using GLA mutational status, researchers suggest. The study, “Mutations ... Read more

October 16, 2018October 16, 2018

Antioxidants May Help Prevent Damage to Heart, Kidneys in Fabry Patients, Research Suggests

News

Inhibition of oxidative compounds using pharmacological or nutritional approaches, in addition to enzyme replacement therapy, may benefit patients with Fabry disease, research suggests. Treatment with antioxidant agents may help ... Read more

July 31, 2018July 31, 2018

Chiesi Acquires Global Development, Sales Rights to PRX-102 as Treatment for Fabry Disease

News

Chiesi, an Italian pharmaceutical company, has acquired the exclusive rights to develop and commercialize PRX-102 (pegunigalsidase alfa) in the U.S. as an investigative therapy for Fabry disease. The agreement expands ... Read more