Please provide your email address to receive an email when new articles are posted on . Wainua has received regulatory approval for polyneuropathy of hereditary transthyretin-mediated amyloidosis.

A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with ...

The US Food and Drug Administration (FDA) has approved vutrisiran (Amvuttra, Alnylam Pharmaceuticals) for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in ...

University College London's National Amyloidosis Center leads a multinational team reporting that a single infusion of an in vivo gene-editing therapy (nexiguran ziclumeran) produced rapid, deep, and ...

WILMINGTON, Del.--(BUSINESS WIRE)--Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed ...

Vutrisiran (Amvuttra) has been approved by the European Commission (EC) for treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or 2 polyneuropathy, the company ...

Credit: Getty Images. Eplontersen is an investigational ligand-conjugated antisense medicine designed to reduce the production of TTR protein to treat both hereditary and nonhereditary forms of ATTR.

WILMINGTON, Del.--(BUSINESS WIRE)-- Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed ...

The outlook for hATTR-PN can vary depending on the genetic mutation involved and how the condition first presents. Newer treatments are helping improve outcomes. hATTR is a rare, hereditary disease. A ...

Based on Nobel Prize-winning CRISPR/Cas9 gene editing technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate ...