hATTR-PN

Scientist Jonathan Wall, PhD, has won a $1.79-million grant to develop a potential treatment for amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). The grant, from the National Institute of Diabetes and Digestive and Kidney Diseases, will fund a research project, titled “Developing a Theranostic…

Ionis Pharmaceuticals has launched a new grant program to fund novice researchers investigating transthyretin (ATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP), also known as hereditary ATTR. “We are proud to establish the Janice Wiesman Young Investigator Grant program, an important initiative to…

Leading an association that supports and advocates for rare disease patients while being a caregiver to one certainly has shaped my outlook on life. To a larger extent, these roles have allowed me to find my calling within the campaign space, where I’m pushing for positive changes to the present…

The symptoms of familial amyloid polyneuropathy (FAP) may make it harder to enjoy a good night’s sleep. To help you sleep better, in addition to medical interventions, you may want to try sleep hygiene techniques. FAP and sleep FAP leads to damage of the nervous system and can cause peripheral…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

Intellia Therapeutics anticipates reporting interim data this year from a Phase 1 study evaluating the safety and activity profile of NTLA-2001, its investigational gene-editing therapy to treat hereditary transthyretin amyloidosis with polyneuropathy, also known as…

Familial amyloid polyneuropathy (FAP) is a rare genetic disorder caused by the buildup of abnormal protein clumps called amyloids throughout the body. These amyloids can affect several parts of the body, including the heart and the nervous system. The information that follows may help you understand how FAP causes…

People living with hereditary transthyretin (hATTR) amyloidosis — which includes familial amyloid polyneuropathy (FAP) — report “profound” life changes due to their disease, particularly as it progresses and affects them physically, emotionally, and socially, a study based on patient interviews reported. These findings emphasize the need for better care…

One of my greatest accomplishments in life is being a mother to four amazing individuals. I cannot give enough credit to my kids for handling all of the challenges that come with having a father stricken with hereditary amyloidosis. When my husband, Aubrey, was diagnosed in 2013, breaking…

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 a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…