Although less able to form damaging protein deposits, the E61K variant of transthyretin appears to cause familial amyloid…
Articles by Joana Carvalho, PhD
Blood levels of neurofilament light chain (NfL) — a protein commonly used as a marker of nerve cell death…
Alnylam Pharmaceuticals announced a potential new biannual dosing regimen for vutrisiran, its investigational treatment for familial amyloid…
The Portuguese Medicine Regulatory Authority, known as Infarmed, has added Tegsedi (inotersen) — an approved treatment for…
Spain’s Ministry of Health has added Tegsedi (inotersen), an injection treatment for stage 1 or 2 polyneuropathy in adults…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to Alnylam Pharmaceuticals’ vutrisiran as a…
Sudoscan, a device that evaluates sweat gland function, may help physicians improve the diagnosis of familial amyloid polyneuropathy…
Akcea Therapeutics and Ionis Pharmaceuticals announced the launch of a Phase 3 clinical trial evaluating the safety and…
Akcea Therapeutics’ next-generation antisense medicine AKCEA-TTR-LRx (ION-682884) — currently in development for…
Tegsedi (inotersen) and Onpattro (patisiran) are both effective treatments to…