Updates
-
-
HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access ... Read more -
Alnylam Opens ‘Advocacy for Impact’ Grant Program Supporting FAP, Like Patient Groups
Alnylam Pharmaceuticals has opened its second annual competitive grants program, supporting patient advocacy projects worldwide that aim to address the unmet and diverse needs of ... Read more -
How to Manage Neuropathy Symptoms During Thanksgiving
Thanksgiving is a time for friends and family to spend together reflecting on the things they’re grateful for and bonding over the traditional meal. ... Read more -
Akcea and Ionis Launch Phase 3 Trial of AKCEA-TTR-LRx in People With FAP
Akcea Therapeutics and Ionis Pharmaceuticals announced the launch of a Phase 3 clinical trial evaluating the safety and efficacy of AKCEA-TTR-LRx (ION-682884) for the ... Read more -
Gait Tracking Using RGB-D Camera Could Aid Doctors in Diagnosing, Managing FAP, Study Shows
An affordable, portable, and non-invasive human motion tracking system based on an RGB-D camera can be useful for monitoring gait impairments in people with ... Read more
Subscribe to our mailing list
Get regular updates delivered to your email box.
Thanks! Please check your email for a confirmation email.
-
Angel loved growing up in a big, close-knit family, surrounded by cousins, aunts and ...
-
Rare Disease Film Festival Highlights Patient and Researcher Unity
Rare disease-themed videos glowed on a large screen before an audience of people in ... -
Therapy Dogs Offer Benefits for Neuropathy Patients
Pets can provide companionship and help with basic tasks to neuropathy patients and their ... -
Protein Modification May Lead to Heart Problems in FAP by Promoting Aggregation of Mutant TTR, Study Says
The addition of the amino acid homocysteine to the mutated transthyretin (TTR) protein found ... -
Starting an Exercise Routine While Managing Peripheral Neuropathy Symptoms
For people who have peripheral neuropathy symptoms associated with familial amyloid polyneuropathy, self-care may ... -
Study Describes Characteristics of Autonomic Symptoms in Hereditary Amyloidosis
Autonomic symptoms are common among patients with hereditary ATTR amyloidosis, usually accompany the progression ...
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