Rare Case of Spinal Canal Narrowing Due to Amyloid Buildup Reported in FAP Patient
Narrowing of the spinal canal due to a buildup of proteins — transthyretin (TTR) amyloid deposits — was reported in a rare case of ... Read more
Updates
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How New Caregivers Must Address Feelings of Denial
The diagnosis of a disease like familial amyloid polyneuropathy in a loved one has a profound impact on the daily life of a new caregiver. ... Read more -
Vyndaqel Safe But Generally Ineffective at Delaying FAP Neuropathy in Long Term, Study Suggests
Long-term treatment with Vyndaqel (tafamidis) is safe and well-tolerated by patients with transthyretin familial amyloid polyneuropathy (TTR-FAP) caused by Val30Met and other mutations, but does ... Read more -
Navigating the Turbulent Feelings of Caregiving
The onset of familial amyloid polyneuropathy forces daunting shifts in the relationships between caregivers and their loved one. When my mother-in-law was diagnosed with polyneuropathy, ... Read more
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Longer Wait for Liver Transplant Can Worsen FAP Afterward, Study Finds
Liver transplants can significantly prolong survival in familial amyloid polyneuropathy (FAP) patients, and the ... -
FAP Patient with Rare Mutation and Advancing Nerve, Heart Damage Treated Ably with Vyndaqel, Case Study Reports
A 60-year-old man with familial amyloid polyneuropathy (FAP), and quickly progressing nerve damage and ... -
Implanting Pacemaker Before Liver Transplant Does Not Lead to Better Outcomes, Study Reveals
Although most patients with familial amyloid polyneuropathy (FAP) who undergo liver transplant will require ... -
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will ... -
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn ... -
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
Rare diseases affect about 30 million Americans — roughly the same number as those ...
Tafamidis Delays Disease Progression in Patients with Transthyretin Amyloid Polyneuropathy, Pfizer Study Suggests
News
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