FDA Approves New Agent for Nerve Pain From Rare Inherited Disease
Eplontersen (Wainua) was approved by the FDA to treat polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (ATTR) in adults, Ionis Pharmaceuticals and AstraZeneca announced on Thursday. Hereditary ATTR is caused by a mutation in the TTR gene that results in the buildup of misfolded transthyretin protein, leading to amyloid deposits in the peripheral nerves (ATTR-PN) or […]
Full-On Reversal of Cardiac Amyloidosis Possible With Antibodies
It is possible for transthyretin amyloid cardiomyopathy (ATTR-CM, also called cardiac ATTR amyloidosis) to be reversed with antibody treatment, as suggested by rare cases of patients who had their ATTR-related heart failure resolve spontaneously, with cardiac structure and function restored to almost normal. In a small case series, three men (ages 68, 82, and 76) […]