News

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…

MMP-14 Levels May Serve as Biomarker and Therapeutic Target in FAP Patients

Matrix metalloprotease-14 (MMP-14) is a novel biomarker for therapy follow-up, as well as a potential therapeutic target in familial amyloidotic polyneuropathy (FAP), said researchers in Portugal. Their study, “MMP-14 overexpression correlates with the neurodegenerative process in familial amyloidotic polyneuropathy,” appeared in the journal Disease Models and Mechanisms. FAP…

EMA Regulatory Committee Grants Accelerated Assessment to Patisiran to Treat FAP

Alnylam Pharmaceuticals’ lead investigational therapeutic patisiran, for the treatment of hereditary ATTR (hATTR) amyloidosis, also known as familial amyloid polyneuropathy (FAP), was granted accelerated assessment by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). “We are pleased the CHMP has granted accelerated assessment for patisiran…

Inotersen, Potential FAP Treatment, Under Review for Approval in Europe

Ionis Pharmaceuticals has asked the European Medicines Agency (EMA) to approve its lead drug candidate inotersen to treat hereditary TTR amyloidosis (ATTR), also known as familial amyloid polyneuropathy (FAP). The request, known as a Marketing Authorization Application (MAA), is supported by positive data from the Phase 3 NEURO-TRR trial, and…