Medically reviewed by Jeffrey S. Lander, MD ATTR-CM is an acronym for the medical condition called transthyretin amyloid cardiomyopathy. There are two causes of ATTR-CM, an inherited gene mutation and ...

It’s a result of a change in a gene, also called a mutation. The mutated TTR gene in the liver makes abnormal shapes of a protein called transthyretin. ... hATTR amyloidosis genetic testing.

All had changes in their TTR gene. About 80% could walk without help (stage 1 nerve damage) and 20% could walk with help (stage 2 nerve damage). A total of 24 people got inotersen.

Voyager Therapeutics presents data on tau silencing therapy VY1706 and anti-amyloid therapy at ASGCT 2025. Quiver AI Summary. Voyager Therapeutics, Inc. has announced that it will present eight ...

Scientists are just beginning to understand the microbes that have been with us for millennia — the so-called microbiome, ...

The Christchurch mutation, which protects against tau pathology and cognitive deterioration despite extensive amyloid buildup, offers an important clue. This rare mutation is found in the APOE gene ...

A new dosing regimen for Kisunla, to help lower the risk of certain side effects, has been approved by the U.S. Food and Drug ...

Amyloidosis is typically thought of as a rare disease, but the variety of subtypes that involve the heart — one of the organs most commonly involved — challenge this characterization ...

Of the 258 participants, 195 were cognitively asymptomatic, 34 had mild cognitive impairment, 23 had dementia, and six had an ...