Apr. 24 at 12:14 PM
$PLX Fabry Disease newsletter: https://fabrydiseasenews.com/news/monthly-elfabrio-treatment-keeps-fabry-disease-stable-5-years/
"The BRIGHT trial showed that this schedule was generally safe and effective in 30 patients who had been on other replacement therapies. Of the 30 patients, 29 enrolled in the open-label extension study to continue receiving Elfabrio for up to an additional seven years. One patient withdrew due to fatigue, leaving 28 continuing patients who have received Elfabrio for three to five years. The total durations are different because patients joined the extension study at different times.
While these results suggest that extending the dosing interval of Elfabrio to every four weeks is feasible and effective for many patients, “the final results of this extension study will further assess the feasibility of this dosing regimen,” the researchers wrote."
