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.

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 ...

Hereditary transthyretin amyloidosis (hATTR) is a rare inherited disorder that gets worse over time. It often affects the nerves, heart, liver, and kidneys, but it can also affect other organs.

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 ...

CARLSBAD, Calif., Dec. 21, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (IONS) (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved Ionis and AstraZeneca's ...

A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin amyloidosis with polyneuropathy. Polyneuropathy stages and disability scores were ...

− Phase 3b Open-Label Study Showed Treatment with Patisiran Achieved Rapid and Sustained Reduction in Serum TTR Levels in hATTR Amyloidosis Patients with Polyneuropathy Progression Following ...

"Our data support the use of sNfL in screening TTRv carriers and in monitoring disease progression and effect of treatment in ATTRv amyloidosis patients…Larger studies are warranted to confirm and ...

WILMINGTON, Del.--(BUSINESS WIRE)-- Eplontersen has been granted Orphan Drug Designation (ODD) in the US by the Food and Drug Administration (FDA) for the treatment of transthyretin-mediated ...

WILMINGTON, Del.--(BUSINESS WIRE)--AstraZeneca and Ionis’ WAINUA™ (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in ...

Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) can affect many parts of the body. Nerve symptoms are often the earliest signs of this condition. hATTR-PN is a rare genetic ...