AT-01, an imaging agent being developed by Attralus, can help to differentiate between transthyretin (ATTR) amyloidosis — which includes familial amyloid polyneuropathy (FAP) — and other forms of amyloidosis, new clinical trial data indicate. The findings were presented in a poster, “Detection of Systemic AL…
Vutrisiran, a second-generation RNA interference (RNAi) therapy candidate, safely and effectively reduces neurologic impairment and improves physical function and quality of life in adults with familial amyloid polyneuropathy (FAP). That’s according to top-line, nine-month data from the global HELIOS-A Phase 3 trial. Alnylam Pharmaceuticals, Vutrisiran’s developer, plans…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
Benzbromarone (BBM), a medicine used to treat gout, can stabilize the TTR protein to the same degree as therapies used to treat people with familial amyloid polyneuropathy (FAP), a study showed. These findings support the further development of…
The case of a woman of Afro-Caribbean descent with early onset transthyretin (TTR) amyloidosis, who was found to carry a TTR mutation more commonly seen in those of Celtic descent, showed that this disorder can be more varied than previously known. Titled “Early-Onset of Transthyretin Amyloidosis in…
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…
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…
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…