Small vesicles in the blood contribute to the clumping or aggregation, and toxic accumulation of the transthyretin (TTR) protein in…
Articles by Marta Figueiredo, PhD
A single dose of NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy, safely and effectively leads to rapid and sustained reductions…
The toxic transthyretin (TTR) clumps that form in people with familial amyloid polyneuropathy (FAP) caused by Val30Met, the most…
An annual decline in sensory nerve function — one greater than 25% — occurs as long as two years before…
The benefits of Vutrisiran in reducing neurologic impairment and overall disability, and improving the quality of life in adults…
The European Medicines Agency (EMA) has agreed to review Alnylam Pharmaceuticals’ application requesting the approval of vutrisiran, its second-generation…
Long-term treatment with Tegsedi (inotersen) helps preserve quality of life in people with familial amyloid polyneuropathy (FAP) for…
Patient enrollment is complete for the Phase 3 NEURO-TTRansform trial testing Ionis Pharmaceuticals’ eplontersen in adults with familial…
A single dose of Intellia Therapeutics’ investigational CRISPR/Cas9-based gene-editing therapy NTLA-2001 safely results in a rapid and strong…
Attralus’ imaging agent AT-01 was able to detect amyloid deposits in the heart, even in patients without heart…