News

Japanese experts have developed guidelines for treating people in their country with transthyretin familial amyloid polyneuropathy that are different from other countries’ guidelines. That’s because…

While Vyndaqel (tafamidis) has been shown to improve symptoms of familial amyloid polyneuropathy (FAP), it likely does not protect against anemia — a common problem…

Alnylam Pharmaceuticals and Sanofi have dissolved the partnership they formed to develop and market two treatments for the neurological disease familial amyloid polyneuropathy, or FAP. The change…

The U.S. Food and Drug Administration (FDA) has agreed to speedily review Ionis Pharmaceuticals’ application to get  its investigational drug inotersen approved as a…

The artificial induction of joint ossification, or surgical arthrodesis, is a safe and effective option to treat patients with rare knee structural deformities due…

A combination of three tests that measure upper limb nerve damage and sensitivity to stimuli could help doctors distinguish between transthyretin familial amyloid polyneuropathy (TTR-FAP) and…

An assessment of the global prevalence of familial amyloid polyneuropathy, caused by mutations in the transthyretin (TTR) gene, gave a similar count to that estimated…

Alnylam Pharmaceuticals and its partner Sanofi Genzyme have submitted an application that asks European regulators to approve patisiran for adults with hereditary transthyretin-mediated…

Alnylam Pharmaceuticals has submitted a complete new drug application (NDA) for its investigational therapy patisiran to the U.S. Food and Drug Administration for the…

The U.S. Food and Drug Administration has agreed to Alnylam Pharmaceuticals’ request to expand the orphan drug designation for patisiran to include a second…