A single dose of an RNA-based gene therapy for Fabry disease produced long-lasting levels of the missing key enzyme and reduced the toxic buildup of fatty molecules linked to the disease, according to new preclinical data from Addition Therapeutics. The findings were presented in a poster at the…
Discussion
Receiving a medical diagnosis can change a person’s life in ways that go far beyond the physical symptoms of a disease. Whether the diagnosis is common or rare, chronic or progressive, the emotional and psychological impact can be profound. For many people, the journey after diagnosis becomes not only a…
Discussion
There is a story that brings 5 million visitors each year to a hamlet nestled in the foothills of the majestic Pyrenees. Lourdes, France, has a population of only about 15,000, but massive crowds of pilgrims have flooded the town since 1858, when news spread of an apparition…
The U.S. Food and Drug Administration (FDA) has agreed to meet with Glafabra Therapeutics to discuss plans for clinical testing of GT-GLA-S03, the company’s gene therapy candidate for Fabry disease. The face-to-face meeting, scheduled for July 16, was arranged through the FDA’s INTERACT program, which allows certain developers…
Discussion
Because having Fabry disease means unexpected things often happen to me, I always have fresh topics to write about. Recently, I’ve experienced several odd occurrences. While I was asleep on the night of April 23, I felt a severe pain in my right calf and heel. Half awake,…
A team of scientists in Spain has developed a new experimental nanoparticle-based treatment approach that aims to address the root cause of Fabry disease through two distinct biological pathways. The new nanomedicine aims to deliver a working copy of the GLA gene, while also reducing production of Gb3, a…
A new experimental gene therapy may boost the production of alpha-galactosidase A (alpha-Gal A) — the enzyme that’s missing or faulty in Fabry disease — beyond the levels of gene therapies now in development. That’s according to laboratory data from a preclinical study that tested the new gene therapy…
Becoming educated and informed about Fabry disease is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your Fabry disease journey.
There is no cure for Fabry disease yet, but there are treatments that can help manage the disease. Learn more about the approved and experimental therapies here.
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