The U.S. Food and Drug Administration (FDA) has advised Sangamo Therapeutics that positive results from a single well-controlled clinical trial, in addition to existing evidence, may be sufficient to form the basis for approving its Fabry disease gene therapy candidate. For Sangamo, that means that one…
The investigational gene therapy ST-920 (isaralgagene civaparvovec) has been well tolerated so far among adults with Fabry disease in the Phase 1/2 STAAR clinical trial, according to new interim data from its developer Sangamo Therapeutics. One-year data from more than a dozen…
Among people receiving medical care for chronic kidney diseases, a small fraction have Fabry disease, a new study highlights. To ensure these patients get optimal treatment, it’s critical to have screening programs in place to provide a prompt diagnosis — especially early on — according to researchers. “Although the…
Over the past two decades, kidney disease and urinary tract problems have become more common among people with Fabry disease, especially among female or Black patients. That’s according to the study, “Prevalence of Kidney and Urinary Tract Complications in Fabry Disease from 2000 to 2020: A…
The first Fabry disease patient has been dosed in a Phase 2 clinical trial that’s testing the experimental oral therapy AL01211. The Phase 2 study (NCT06114329) is expected to enroll 18 male patients with classic Fabry disease, ages 18 to 60, who have not received any prior…
A team of expert clinicians and patient advocates has created a new set of recommendations to guide the use of Galafold (migalastat) in people with Fabry disease, highlighting the importance of centering patients’ preferences in treatment decisions. “We hope that this publication will lead to the provision of…
People with Fabry disease who have burning limb pain or depression are more likely to report poor quality of life, a new study shows. Addressing these issues may help them, researchers said in “Screening for health-related quality of life and its determinants in Fabry disease: A…
A gene therapy treatment increased levels of the deficient alpha-glactosidase A (Gal A) enzyme in the organs of mice in a model of Fabry disease, according to a new study. Results showed the gene therapy approach reduced the toxic buildup of fatty molecules in most organs assessed, but not…
Enzyme replacement therapy (ERT) can help to increase the bone density of male patients with Fabry disease, who tend to have lower bone mineral density, a study has found. Female patients, by contrast, were found generally not to have low bone density, and ERT did not substantially change…
An imaging technology called 3D echocardiography, which allows clinicians to visualize the heart in three dimensions and measure the strain on heart tissue when the heart beats, may be useful for assessing heart damage among people with Fabry disease. That’s according to the study, “Three-dimensional echocardiographic…
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