Researchers have uncovered previously unknown structural features of transthyretin, the protein that’s faulty in familial amyloid polyneuropathy (FAP), which could pave the way toward new treatment strategies. “We’ve unveiled a molecular complexity that has been hidden from researchers for decades, which enables us to design better medicines to stabilize…

Several cases in which treatment with Onpattro (patisiran) was used to ease familial amyloid polyneuropathy (FAP) symptoms were described in a recent report from Italy. The study, “Italian Real-Life Experience of Patients with Hereditary Transthyretin Amyloidosis Treated with Patisiran,” was published in Pharmacogenomics and Personalized…

Protego Biopharma has raised $51 million to support the development of therapies for diseases caused by protein misfolding, such as familial amyloid polyneuropathy (FAP). The series A financing was co-led by Lightspeed Venture Partners, Vida Ventures, and MPM Capital. As part of the financing, representatives from these three…

The addition of the amino acid homocysteine to the mutated transthyretin (TTR) protein found in patients with familial amyloid polyneuropathy may contribute to heart problems in these patients, a recent study suggests. Specifically, this addition destabilizes and promotes the aggregation of mutant TTR protein, but it does not affect the…

Vyndaqel (tafamidis) increases the stability of the transthyretin protein and may help people with familial amyloidosis polyneuropathy (FAP) carrying a TTR gene mutation common in Asian populations, an early study  from Taiwan suggests. …

Variability in the onset and penetrance of TTR-related amyloidosis (ATTR) among members of the same family may be linked to the expression of different copies of the TTR gene throughout the aging process, a study suggests. The study, “Monoallelic expression of the TTR gene as a contributor to…

The effect of the familial amyloid polyneuropathy (FAP) hallmark protein transthyretin (TTR) on cells is associated with its specific three-dimensional shape, according to Italian researchers. Their study, “A FTIR microspectroscopy study of the structural and biochemical perturbations induced by natively folded and aggregated transthyretin in HL-1…

Studying the interaction between different amyloid (abnormal disease-causing) proteins may provide insight into the treatment of diseases such as familial amyloid polyneuropathy (FAP), an Indian study suggests. The study, “Q-Rich Yeast Prion [PSI+] Accelerates Aggregation of Transthyretin, a Non-Q-Rich Human Protein,” appeared in the journal Frontiers in…

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 TTR-FAP manifests itself differently in Japanese than in people elsewhere. It has a broader range of symptoms in Japanese patients, for example. The guidelines are…

Researchers reported a rare case of a patient with type 1 familiar amyloid polyneuropathy (FAP) who went to the emergency room with pain in his right eye, and was diagnosed with a secondary type of glaucoma 14 years after a liver transplant. The patient was treated only…