Elfabrio (pegunigalsidase alfa) is as safe and effective as Fabrazyme (agalsidase beta) and can prevent kidney decline in adults with Fabry disease over two years, according to final data from the BALANCE Phase 3 clinical trial. Infusion-related side effects, in particular, occur significantly less often with Elfabrio than…
The duration and rate of Fabrazyme (agalsidase beta) infusion can be safely changed in people with Fabry disease, independently of their body weight, if appropriate care is taken, according to a new study from Japan. Specifically, an infusion time shorter than 90 minutes or an infusion rate higher…
Enzyme replacement therapy (ERT), which provides the specific enzymes missing in Fabry patients, can help to ease or even stop your symptoms if you have this disease. However, it is a lifelong treatment, with the enzyme given continuously over time. Here is an overview of what to expect if…
Fabrazyme is an approved treatment for Fabry disease patients age 8 and older. It was developed by Sanofi Genzyme. Following are answers to some frequently asked questions about the treatment. How does Fabrazyme work? Fabrazyme is an enzyme replacement therapy. In patients with Fabry…
What is my job as a parent? In thinking this question through, I came up with a few answers, but it’s not an exhaustive list. I am to protect, guide, teach, and love my kids. At some point, the protection I give them will lessen. They will be…
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