Impact of Inotersen on Functioning and Activities of Daily Living for Patients with Hereditary TTR Amyloidosis: Results from a Double-Blind Placebo-Controlled Trial
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DOI: 10.1182/blood-2018-99-119578
Abstract: Background: Hereditary transthyretin amyloidosis (hATTR) is a rare, systemic, progressive, and fatal condition in which misfolded proteins, mainly produced in the liver, deposit in muscle and organ tissues leading to symptoms of peripheral neuropathy, and… read more here.
Keywords: impact; substantial impairment; placebo; consultancy ... See more keywords