Author Archives: Patricia Inacio PhD

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

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…

Three recently approved therapies for treating familial amyloid polyneuropathy (FAP), also known as hereditary transthyretin amyloidosis with polyneuropathy, have won their developers Best Biotechnology Product honors in the 2020 Prix Galien USA Awards, the Galien Foundation announced. Ionis Pharmaceuticals and its subsidiary Akcea Therapeutics claimed the…

Vyndaqel (tafamidis meglumine) remains safe after more than three years of treatment in patients with familial amyloid polyneuropathy (FAP), with no new safety concerns raised, according to an early analysis of a clinical trial in Japan. The study, “Characteristics of Patients with Hereditary Transthyretin Amyloidosis…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

Two FDA-approved treatments for high blood pressure, diuretics called indapamide and hydrochlorothiazide, might also treat diseases associated with amyloid deposits, such as familial amyloid polyneuropathy (FAP), a study suggests. Diuretics are medicines that help a person excrete more water and salt from their system to lower blood…

Alnylam Pharmaceuticals has opened its second annual competitive grants program, supporting patient advocacy projects worldwide that aim to address the unmet and diverse needs of ATTR amyloidosis, a group of conditions that include familial amyloid polyneuropathy (FAP). Applications for the program, called Advocacy for Impact, are now open and can…

Variability in the onset and penetrance of TTR-related amyloidosis (ATTR) among members of the same family may be linked to the expression of different copies of the TTR gene throughout the aging process, a study suggests. The study, “Monoallelic expression of the TTR gene as a contributor to…

A review of 542 cases of hereditary transthyretin amyloid polyneuropathy (ATTR-PN) from more than 100 countries suggests that the clinical symptoms and geographical occurrence of the disease is more variable than described previously. The review study,“Epidemiological and clinical characteristics of symptomatic hereditary transthyretin amyloid polyneuropathy: a global case series,”…

Cardiac problems are the leading cause of death among transthyretin familial amyloid polyneuropathy (ATTR, also called FAP) patients who have had a liver transplant, according to a retrospective longitudinal study. The study, “Cause of death analysis and temporal trends in survival after liver transplantation for transthyretin familial amyloid polyneurophathy,”…