hATTR-PN

PRX004, an investigational treatment by Prothena, can reduce the progression of nerve damage (neuropathy) and improve heart function in people with hereditary transthyretin amyloidosis (ATTR), which includes familial amyloid polyneuropathy (FAP), according to results from a Phase 1 trial. “We are pleased to see evidence of both a slowing…

2020 is almost at an end. In a few weeks, we will celebrate the holiday season, and before we know it, 2021 will be upon us. The past year was mostly filled with bad news and unexpected events, and the turmoil caused by COVID-19 has impacted many people around the…

Living with a chronic disease such as familial amyloid polyneuropathy (FAP) can be overwhelming. The symptoms of FAP, which may include cardiac impairment, infection, extreme weight loss, muscle wasting, and severe peripheral neuropathy, can significantly affect your quality of life and may even lead to depression. The following is…

Abdominal fat pad biopsy is an accurate diagnostic tool for patients with suspected hereditary transthyretin amyloidosis (ATTR), showing the greatest sensitivity in those with ATTR with polyneuropathy, also known as familial amyloid polyneuropathy (FAP). Importantly, findings show, the…

“Surprise!” I watched familiar faces filter into the room where I stood stunned, not expecting the birthday event that was unfolding before me. I turn 50 tomorrow, and some close friends went to the trouble of organizing a celebration for me. I was thankful for the moment, and I felt…

If you or a loved one has familial amyloid polyneuropathy (FAP), massage therapy may help reduce the impact of peripheral neuropathy symptoms and lower the sense of stress that this progressive genetic disease can cause. What is FAP? FAP is a rare disease caused by mutations in the…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

Onpattro (patisiran) results in sustained improvements in polyneuropathy, quality of life, and other symptoms among people with familial amyloid polyneuropathy (FAP), while showing an acceptable safety profile, according to a one-year interim analysis from an open-label extension clinical trial. The study, “Long-term safety and efficacy…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

Onpattro (patisiran) is more effective than Tegsedi (inotersen) at reducing neurological impairment and improving quality of life in people with familial amyloid polyneuropathy (FAP), a comparison of data from two clinical trials reported. The…