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.

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 ...

WASHINGTON – Angel says her family has been living with a rare disease they called “the curse” for generations. Known as hATTR Amyloidosis, the life-threatening condition was first accurately ...

The investigational RNAi therapeutic vutrisiran (Alnylam Pharmaceuticals) met its primary and secondary endpoints in a phase 3 study of adults with hereditary transthyretin-mediated amyloidosis (hATTR ...

In a position statement published in Orphanet Journal of Rare Diseases, experts suggest that understanding the course of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) can be best ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it has achieved full patient enrollment in its APOLLO-B ...

TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Variants in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as ...

(RTTNews) - RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (ALNY) announced Tuesday that the European Commission has granted marketing authorization for AMVUTTRA (vutrisiran), an RNAi ...

(BPT) – In 1982, Rosaline’s Aunt Polly passed away at age 58 from a rare disease called hereditary ATTR (hATTR) amyloidosis after enduring years of debilitating and progressive symptoms. Learning the ...

hATTR is caused by the misfolding of the TTR protein, which results in amyloid fibril deposition in tissues, including that of the heart and gastrointestinal tract. Specifically, heart failure, ...

Living with hATTR-PN can be challenging, both physically and emotionally. However, multiple resources can offer treatment support, financial help, and ways to connect with others. Hereditary ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Committee for Medicinal Products for Human Use (CHMP) ...