News

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials…

Researchers identified a very rare mutation in a Libyan man who was diagnosed with late-onset familial amyloid polyneuropathy (FAP), according to a case report.

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference…

Vyndaqel (tafamidis) significantly delays the progression of familial amyloid polyneuropathy (FAP) and improves the quality of life of FAP patients, a real-world study shows.

Studying the interaction between different amyloid (abnormal disease-causing) proteins may provide insight into the treatment of diseases such as familial amyloid polyneuropathy (FAP), an…

Akcea Therapeutics has completed a deal giving it the global marketing rights to its affiliate  Ionis Pharmaceuticals’ familial amyloid polyneuropathy therapy inotersen. The deal also…

Familial amyloid polyneuropathy (FAP) is linked to important alterations of the autonomic nervous system, which regulates certain body processes such as breathing, heart rate,…

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies…

A rare mutation of a gene involved in late-onset familial amyloid polyneuropathy occurs at higher rates in one Japanese region, a case study reports. The research,…