The U.S. Food and Drug Administration (FDA) has given orphan drug designation to AT-02, Attralus’ investigational candidate for the treatment of transthyretin amyloidosis (ATTR), a group of diseases that includes familial amyloid polyneuropathy (FAP). Orphan drug status supports the development of potential treatments for rare diseases, those…
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Diflunisal is used off-label to treat familial amyloid polyneuropathy (FAP), but it may be as safe and effective as tafamidis free acid, which is approved in the U.S. under the brand name Vyndamax for treating a FAP-related condition, a study in Taiwan suggests. Nearly all the evaluated FAP patients…
Adults with familial amyloid polyneuropathy (FAP) living in Canada may soon be able to receive Amvuttra (vutisiran) at little or no cost through public drug plans, the therapy’s developer said. Amvuttra is approved in the country for adult FAP patients with stage 1 or stage 2 polyneuropathy or…
The Medicines and Healthcare products Regulatory Agency (MHRA) in the U.K. has approved the injection therapy eplontersen — under the brand name Wainzua — for treating adults with familial amyloid polyneuropathy (FAP). Additionally, a European Union regulatory committee has recommended the treatment’s approval in the EU for adults…
About a month ago, my father-in-law came down with what seemed like a cold. But his condition gradually worsened, which sent him to the hospital. There he was diagnosed with COVID-19. Because he takes on most of the caregiving duties for my mother-in-law, who has familial amyloid polyneuropathy,…
My late husband, Aubrey, was diagnosed with hereditary ATTR amyloidosis in 2013 and passed away on May 22, 2022. Those nine years were spent transitioning from having harmony in our marriage to navigating a relationship wrought with challenges. We were encumbered not only by the stress of Aubrey’s…
Becoming educated and informed about FAP 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 FAP journey.
There is no cure for FAP yet, but there are treatments that can help manage the disease. Learn more about the approved and experimental therapies here.
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