Author Archives: Marisa Wexler MS

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…

FDA Reviewing Application of PRX-102 as Fabry Treatment

Note: This story was updated Sept. 9, 2020, to reflect that PRX-102 is licensed to Chiesi for all markets, including the U.S. The U.S. Food and Drug Administration (FDA) has accepted for review an application from Chiesi Global Rare Diseases and Protalix BioTherapeutics that asks for approval of…

FDA Puts Fabry Gene Therapy 4D-310 on Fast Track

4D-310, a new gene therapy for Fabry disease that is being developed by 4D Molecular Therapeutics (4DMT), has been granted fast track designation by the U.S. Food and Drug Administration (FDA). This designation is given to investigational therapies that the FDA deems to have…