Hereditary transthyretin (hATTR) amyloidosis is a rare disease that runs in families. It is caused by the buildup of toxic proteins called amyloid fibrils throughout the body. The amyloid fibrils ...
Promising therapies aim to treat hATTR and improve quality of life Medically reviewed by Stella Bard, MD Hereditary transthyretin amyloidosis, or hATTR, is a rare disease that causes proteins to ...
Hereditary transthyretin-mediated amyloidosis (hATTR) is a type of amyloidosis, which is a group of diseases where abnormal proteins (amyloids) build up and damage areas of the body. When the ...
NICE has recommended that Akcea’s hereditary transthyretin-related amyloidosis (hATTR) treatment Tegsedi be made available on the NHS in England, ahead of a rival therapy from Alnylam.
Amvuttra is used in adults to treat polyneuropathy (nerve damage) related to hereditary amyloid transthyretin-mediated (hATTR) amyloidosis. This is a rare genetic (inherited) condition that causes ...
NICE has reversed its stance on Alnylam’s gene-silencing drug Onpattro for rare disease hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), backing NHS funding. hATTR is an ultra ...
Both drugs are approved for polyneuropathy caused by hereditary transthyretin amyloidosis (hATTR-PN), with Onpattro’s FDA nod coming in 2018. Alnylam's next-gen RNA drug Amvuttra wins FDA ...
Vutrisiran – an RNAi therapeutic marketed in various countries globally as a treatment of adults with hATTR amyloidosis with polyneuropathy, and in development for the treatment of adults with ...