While Vyndaqel (tafamidis) has been shown to improve symptoms of familial amyloid polyneuropathy (FAP), it likely does not protect…
Articles by Magdalena Kegel
Alnylam Pharmaceuticals and Sanofi have dissolved the partnership they formed to develop and market two treatments for the neurological disease familial amyloid…
The U.S. Food and Drug Administration (FDA) has agreed to speedily review Ionis Pharmaceuticals’ application to get its investigational…
An assessment of the global prevalence of familial amyloid polyneuropathy, caused by mutations in the transthyretin (TTR) gene, gave a…
Alnylam Pharmaceuticals and its partner Sanofi Genzyme have submitted an application that asks European regulators to approve patisiran…
Alnylam Pharmaceuticals has submitted a complete new drug application (NDA) for its investigational therapy patisiran to the U.S. Food…
Researchers have found that a large proportion of untreated people with the TTRVal30Met mutation — causing familial amyloid polyneuropathy (FAP) — have…
Alnylam Pharmaceuticals has started its submission of a rolling new drug application (NDA), asking the U.S. Food and Drug…
Alnylam Pharmaceuticals, together with its partner Sanofi Genzyme, has now released the full results from the Phase 3…
Patients with familial amyloid polyneuropathy (FAP) appear to have intact reproductive capacity, even when they have undergone a liver transplant…