2013 was a landmark year for my husband, Aubrey, and me. That October, we received confirmation that Aubrey has hereditary ATTR amyloidosis. The results were ... Read more
Weight Loss, Other GI Symptoms, Common in FAP, Italian Study FindsGastrointestinal (GI) symptoms, particularly unintentional weight loss, occur frequently in people with hereditary transthyretin amyloidosis, also known as familial amyloid polyneuropathy (FAP), according to data ... Read more
Letting Go of Expectations Helps Me Embrace My Husband’s ChangesSince my husband, Aubrey, was diagnosed with hereditary ATTR amyloidosis in 2013, my journey as his caregiver has taken me to some difficult places. The ... Read more
European Commission Grants NTLA-2001 Gene-editing Therapy Orphan Drug StatusThe European Commission (EC) has granted orphan drug status to Intellia Therapeutics’ NTLA-2001 gene-editing therapy for transthyretin amyloidosis (ATTR), a group of disorders that also ... Read more
Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation FindsAs anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how ... Read more
Subscribe to our mailing list
Get regular updates delivered to your email box.
Thanks! Please check your email for a confirmation email.
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 the ... Read more