News

Intellia Therapeutics anticipates reporting interim data this year from a Phase 1 study evaluating the safety and activity profile of…

People living with hereditary transthyretin (hATTR) amyloidosis — which includes familial amyloid polyneuropathy (FAP) — report “profound” life changes due to their disease, particularly…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient…

In focus groups, transthyretin (ATTR) amyloidosis patients and families reported challenges and concerns related to the odyssey of getting a diagnosis,…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by…

The levels of blood GDF-15 could detect the development and progression of heart disease in people with hereditary transthyretin (ATTRv) amyloidosis, which includes familial…

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening…

Investigating how plasma from people with transthyretin (ATTR) amyloidosis affects heart cells from rats may help researchers predict future heart-related health events and deaths in…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that…