It is a summer morning here in New Zealand. As I write this, a cool, gentle breeze blows through the house, and I am ... Read more
Vutrisiran Reduces Neurologic Damage, Improves Physical Function, Trial Data ShowVutrisiran, a second-generation RNA interference (RNAi) therapy candidate, safely and effectively reduces neurologic impairment and improves physical function and quality of life in adults ... Read more
Searching for Peace Despite Chronic Sleep DeprivationSleep is an essential part of life that comes so naturally, from the moment we come into being. Yet sometimes we forget the impact ... Read more
NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ ForumThe National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual ... 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