Levels of calprotectin, a marker of inflammation, are elevated in the feces of people with familial amyloid polyneuropathy (FAP) who experience gastrointestinal (GI) symptoms, ... Read more
Making the Most of Your Doctor’s Visit When You Have FAPManaging your treatment and care when you have a rare disease such as familial amyloid polyneuropathy (FAP) can be challenging, especially if your care ... Read more
FAP Treatment Tegsedi Approved for Reimbursement in ItalyThe Italian Medicines Agency has added Tegsedi (inotersen), a treatment for familial amyloid polyneuropathy (FAP), to the list of reimbursed therapies in the country. ... Read more
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During PandemicThe Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization ... Read more
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Tafamidis Delays Disease Progression in Patients with Transthyretin Amyloid Polyneuropathy, Pfizer Study Suggests
An interim analysis of the Phase 3 trial for tafamidis (Fx-1006A) as a therapy for patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP) shows the investigational treatment delays disease progression, Pfizer announced. The study, “Long-term safety and efficacy of tafamidis for ... Read more