Author Archives: Marisa Wexler MS

Protalix Asks FDA to Discuss Future of PRX-102

Protalix BioTherapeutics has requested a type A meeting with the U.S. Food and Drug Administration (FDA) to discuss a path forward for approval of PRX-102 (pegunigalsidase alfa), an investigational therapy for Fabry disease that was rejected by the regulatory agency earlier this year. The meeting is expected…

Experts Review Benefits, Challenges of Family Genetic Testing

Family genetic testing can help to diagnose people with rare genetic conditions like Fabry disease, but there still are several barriers to implementing family genetic testing worldwide, according to an international panel of researchers who are using their experiences to share strategies for improving such testing. The review, “…

New Dosing Protocol May Shorten Fabrazyme Infusions

A new infusion protocol that gradually reduces the amount of time needed for the delivery of the Fabry disease therapy Fabrazyme (algasidase beta) is safe and well-tolerated, a study reports. The study, “Stepwise shortening of agalsidase beta infusion duration in Fabry disease: Clinical experience with infusion…

New Study Explores Variability in ERT Outcomes

Enzyme replacement therapy (ERT) may not effectively manage all aspects of Fabry disease in all patients, particularly when started at more advanced ages, a new study indicates. The findings highlight the need to start treatment early, its researchers said. The study, “Variable clinical features of patients with…

Ultrasound for Renal Cysts May Hasten Diagnosis

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 such cysts could aid in the diagnosis of Fabry. The study, “Renal Ultrasound contributes to Fabry…

Charitable Program by Takeda Helps Patients Worldwide Gain Treatment

A charitable program can help people with lysosomal storage disorders, such as Fabry disease, who live in underserved communities worldwide gain access to essential treatments, a study reports. The study, “A charitable access program for patients with lysosomal storage disorders in underserved communities worldwide,” was published in…