Small vesicles in the blood contribute to the clumping or aggregation, and toxic accumulation of the transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP), a study shows. These findings shed light on the underlying mechanisms of FAP and point to these small blood vesicles, known as extracellular…
A single dose of NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy, safely and effectively leads to rapid and sustained reductions in the levels of the damaging transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP), according to interim data from a Phase 1 trial. Notably, maximum TTR reductions…
The toxic transthyretin (TTR) clumps that form in people with familial amyloid polyneuropathy (FAP) caused by Val30Met, the most common disease-causing mutation, are structurally different in the eye and the heart, a study suggests. These findings highlight the structural variability of TTR clumps between organs, even when associated with…
An annual decline in sensory nerve function — one greater than 25% — occurs as long as two years before the onset of familial amyloid polyneuropathy (FAP) in people carrying Val30Met, the most common disease-causing mutation in FAP, a study shows. Such drops in function, as measured by non-invasive…
The benefits of Vutrisiran in reducing neurologic impairment and overall disability, and improving the quality of life in adults with familial amyloid polyneuropathy (FAP) were sustained over 18 months, according to new data from the ongoing Phase 3 HELIOS-A trial. “These results build on the positive vutrisiran data…
The European Medicines Agency (EMA) has agreed to review Alnylam Pharmaceuticals’ application requesting the approval of vutrisiran, its second-generation RNA interference (RNAi) therapy candidate for familial amyloid polyneuropathy (FAP). Hereditary ATTR (hATTR) amyloidosis, which includes FAP, “is a rare, rapidly progressive, debilitating and fatal condition and we are…
Long-term treatment with Tegsedi (inotersen) helps preserve quality of life in people with familial amyloid polyneuropathy (FAP) for up to three years, according to data from the Phase 3 NEURO-TTR trial and its extension study. While patients initially assigned to a placebo stopped experiencing declines in quality of…
Patient enrollment is complete for the Phase 3 NEURO-TTRansform trial testing Ionis Pharmaceuticals’ eplontersen in adults with familial amyloid polyneuropathy (FAP). The trial exceeded its enrollment target of 140 participants, with Ionis now expecting to register more than 160 patients in the study. “The speed of enrollment…
A single dose of Intellia Therapeutics’ investigational CRISPR/Cas9-based gene-editing therapy NTLA-2001 safely results in a rapid and strong reduction in the levels of the damaging transthyretin (TTR) protein, according to interim data from the first six familial amyloid polyneuropathy (FAP) patients given the therapy in a Phase…
Attralus’ imaging agent AT-01 was able to detect amyloid deposits in the heart, even in patients without heart symptoms and with normal levels of a heart damage biomarker, according to data from a Phase 1/2 clinical trial. The imaging agent was also able to distinguish between patients with…