One of my greatest accomplishments in life is being a mother to four amazing individuals. I cannot give enough credit to my kids for ... Read more
Rare Disease Day ‘Patient Hero’ Raises Awareness Through FashionDiagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from ... Read more
Rare Disease Day at NIH, Set for March 1, Growing Year by YearRare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, ... Read more
Focus Groups Highlight Need for Patient Input in Diagnosis, TreatmentIn focus groups, transthyretin (ATTR) amyloidosis patients and families reported challenges and concerns related to the odyssey of getting a diagnosis, the effect of ... 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