News

Vutrisiran — now known by the brand name Amvuttra — has been approved by the U.S. Food and Drug Administration (FDA) to treat familial amyloid polyneuropathy (FAP), also known as hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy. The therapy is expected to be available for shipment to healthcare providers…

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…

Vutrisiran continues to show promise at lessening heart damage in adults with familial amyloid polyneuropathy (FAP) with cardiac involvement, according to exploratory new data from the ongoing Phase 3 HELIOS-A trial. Data collected over 18 months of treatment in patients with pre-existing evidence of cardiac involvement showed that vutrisiran led…

Fat accumulation in the soleus muscle, a large muscle of the lower leg, may be indicative of disease progression in people with hereditary transthyretin (ATTRv) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP), a small study in China suggests. The study, “Skeletal Muscle Involvement Pattern of…

A seven-year genetic screening program in Bulgaria involving people at high risk of hereditary transthyretin amyloidosis — a group of conditions that also includes familial amyloid polyneuropathy (FAP) — found patients showed mixed symptoms, with some clinical peculiarities related to specific disease-causing mutations. According to researchers, such genetic screening is…

The first patient has been dosed in the second part of a Phase 1 trial evaluating NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis. The first part of the study exposed patients to single ascending doses of…

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

A genetic variant in TTR — the gene implicated in familial amyloid polyneuropathy (FAP) — was linked with a greater risk of heart failure and death among Black people living in the U.S. in a recent study. In fact, being a carrier of the specific…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

In a real-world study of patients with worse disease severity at the start of treatment, Tegsedi (inotersen) slowed the progression of hereditary transthyretin amyloidosis — a group of disorders that also includes familial amyloid polyneuropathy (FAP) — and preserved quality of life. As progression is associated with disease…