Iqra Mumal, MSc,  —

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Tegsedi (inotersen) as a treatment for Stage 1 or 2 polyneuropathy in adults with hereditary transthyretin amyloidosis (hATTR), also known as familial amyloid polyneuropathy (FAP). The announcement comes from Akcea Therapeutics and…

Cold and pinprick sensitivity in the hands and electrical impulse speed in nerves can help doctors make an early diagnosis of transthyretin familial amyloid polyneuropathy (TTR-FAP), a study reports. The research, “Transthyretin familial amyloid polyneuropathy (TTR-FAP): Parameters for early diagnosis,” appeared in the journal Brain & Behavior. Transthyretin…

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…

The discovery of serum proteins that are specific to FAC (familial amyloid cardiomyopathy) and FAP (familial amyloid polyneuropathy) may aid in the diagnosis and prognosis of these diseases, new research shows. The study, “Serum Proteomic Variability Associated with Clinical Phenotype in Familial Transthyretin Amyloidosis (ATTRm),” was published in…

A new study outlines the design and rationale for APOLLO, a Phase 3 clinical trial (NCT02510261) to evaluate the effect of patisiran (ALN-TTR02) in patients with hATTR (hereditary ATTR) amyloidosis with polyneuropathy. The study, “Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in…

Researchers presented the main features of primary amyloidosis, showing the need for careful diagnosis in patients that present with neuropathic symptoms, especially autonomic neuropathy, to correctly identify other conditions such as familial amyloid neuropathy (FAP). The study, titled “Autonomic Neuropathy and Albuminocytologic Dissociation in Cerebrospinal Fluid As the Presenting…

An imaging technique that uses tiny amounts of radioactive chemicals can help doctors predict the outcomes of cases of familial amyloid polyneuropathy, according to a Portuguese study. The research also demonstrated that a liver transplant can stabilize FAP patients’ heart conditions. The University of Lisbon study was published in the journal…