Author Archives: Magdalena Kegel

Vyndaqel May Not Protect FAP Patients Against Anemia, Study Finds

While Vyndaqel (tafamidis) has been shown to improve symptoms of familial amyloid polyneuropathy (FAP), it likely does not protect against anemia — a common problem among patients with the disease, a small Japanese study found. The data suggests that physicians may need to pay attention not only to patients’ neurological…

Alnylam and Sanofi Ask European Regulators to Approve Patisiran for FAP

Alnylam Pharmaceuticals and its partner Sanofi Genzyme have submitted an application that asks European regulators to approve patisiran for adults with hereditary transthyretin-mediated (hATTR) amyloidosis. The European Medicines Agency (EMA) announced earlier that it will review the so-called Marketing Authorisation Application (MAA) under an accelerated assessment pathway, which will…

Cognitive Impairment Common Among Older, Untreated FAP Patients

Researchers have found that a large proportion of untreated people with the TTRVal30Met mutation — causing familial amyloid polyneuropathy (FAP) — have cognitive impairment, particularly if they experience disease symptoms. Such problems are more common in older patients and those with late-onset disease, the study, published in the Journal of Neurology…