Following discussions with regulatory authorities in the U.S. and Europe, Idorsia has outlined a new Phase 3 registration program to evaluate its experimental oral therapy, lucerastat, for its effects on kidney outcomes in people with Fabry disease. If the studies are successful, Idorsia…
News
People with Fabry disease have abnormalities in the blood vessels within the eye, and these abnormalities may be indicative of heart disease in Fabry patients, a first-of-its-kind study reports. Researchers also found an association between eye vessel abnormalities and more inflammation in people with certain Fabry-causing mutations…
The experimental gene therapy AMT-191 appears to be working as intended in an early clinical trial, developer uniQure said, but the company is pausing part of the study pending further investigation into severe liver damage cases. “While the study remains ongoing, we believe the preliminary data collected are supportive…
Sangamo Therapeutics has begun seeking accelerated approval from the U.S. Food and Drug Administration (FDA) for isaralgagene civaparvovec (formerly ST-920), its experimental gene therapy for Fabry disease. Accelerated approval is a type of conditional approval in which the FDA allows a therapy to be sold based…
Long-term dosing with lucerastat, an experimental substrate reduction treatment from Idorsia, may slow the loss of kidney function in adults with Fabry disease, perhaps by reducing the buildup of toxic fatty molecules in the body’s cells, a global clinical trial has found. The oral therapy — given…
Markers of disease-related tissue damage in the brains of Fabry disease patients treated with Galafold (migalastat) tend to remain stable over time, a study found. Fabry patients with high blood pressure may be more likely to have damage in the brain, according…
Despite a delayed start until adulthood, enzyme replacement therapy (ERT) was associated with long-term cardiac stability in a man with Fabry disease who had shown symptoms since early childhood, a case study from Brazil shows. Because ERT was initiated before any signs of heart involvement appeared, the man…
Nearly one in five adults with Fabry disease experienced a stroke over roughly a decade of follow-up, with risk shaped by genetic factors, kidney function, and coexisting autoimmune disease, a large U.K. study found. Preserved kidney function and carrying the disease-causing N215S mutation — which is often linked to…
Health Canada has approved the enzyme replacement therapy Elfabrio (pegunigalsidase alfa) to treat adults with Fabry disease. “With Health Canada’s approval of Elfabrio, we’re proud to have a new treatment option available to Canadians living with Fabry disease,” John Hess, senior vice president for the Americas at…
Young adults with Fabry disease in Canada are at a significantly higher risk of experiencing a first-time stroke or transient ischemic attack (temporary blockages sometimes referred to as “mini-strokes”) than the general population, a new study reveals. Despite this elevated risk, very few Fabry patients who experienced a first…
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