News

Vienna to Host RARE2019 Meeting on Rare Diseases

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Enzyme Replacement Therapy Partially Restores Cellular Functions in Patients With Fabry and Gaucher Diseases, Study Finds

Enzyme replacement therapy (ERT) partially restores the function of lysosomes and mitochondria in patients with Gaucher and Fabry diseases, a study finds. The study, “Impaired autophagic and mitochondrial functions are partially restored by ERT in Gaucher and Fabry diseases,” was published in PLOS One. Fabry and Gaucher…

Avrobio Earns FDA Orphan Drug Designation for Fabry Treatment AVR-RD-01

The U.S. Food And Drug Administration (FDA) has granted orphan drug status to Avrobio’s investigational gene therapy AVR-RD-01 for the treatment of Fabry disease. AVR-RD-01 is being tested in two separate clinical trials that are still recruiting patients: AVRO-RD-01-201 Phase 2 trial (NCT03454893) and an investigator-sponsored Phase…

Protalix BioTherapeutics Completes Enrollment for PRX-102 Phase 3 Trial  

Protalix BioTherapeutics announced it has completed enrollment of its Phase 3 trial evaluating PRX-102 in Fabry disease patients previously treated with Shire’s Replagal (agalsidase alfa). The BRIDGE trial (NCT03018730) is an open-label switch-over study that will assess the safety and efficacy of PRX-102 in 22 patients with Fabry disease treated with Replagal for…