Traveling with Nerve Pain Symptoms
The peripheral symptoms of familial amyloid polyneuropathy (FAP) may prove troublesome for the maneuverability of patients. Caregivers are faced with the daunting task of ... Read more
Updates
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Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their ... Read more -
Late-onset FAP Associated with Misdiagnosis, Greater Severity, Brazilian Study Finds
Familial amyloid polyneuropathy (FAP) patients carrying the Val30Met mutation are more frequently misdiagnosed and tend to have neurologic and cardiac complications more commonly if ... Read more -
Variants in C1QA and C1QC Genes Associated with Age at Onset in Portuguese Families
Variants in two genes involved in the body’s immune response and inflammation were associated with age-of-onset of symptoms in Portuguese families with familial amyloid ... Read more
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Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims ... -
Here’s How Caregivers Can Adapt to the Acceptance Stage of Grief
The diagnosis of a rare disease in a loved one is enough to usher ... -
#AANAM – Tegsedi Continues to Benefit FAP Patients for up to Two Years
Tegsedi (inotersen) continues to slow progression of familial amyloid polyneuropathy (FAP) and maintains the ... -
New Caregivers Should Be Proactive When Dealing with Depression Risk
For new caregivers, depression is a clear and present danger. As the fourth of ... -
Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
With 250 rare diseases newly identified every year, scientists can barely keep up — ...
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