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APOLLO-B study impact
26 October 2023
"Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis"
This is the first trial to show the clinical benefit of TTR knockdown (via RNA interference) in ATTR amyloid cardiomyopathy. Whilst RNAi was approved in 2018 for the treatment of hereditary amyloid polyneuropathy, the benefits in patients with ATTR amyloid cardiomyopathy, a far more common condition, were yet to be established until the current study. This trial opens up a completely new treatment paradigm for patients with this increasingly diagnosed cause of heart failure.
Maurer, M. S., et al. (2023). "Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis." New England Journal of Medicine 389(17): 1553-1565.
Professor Mariannna Fontana, Department of Inflammation, Division of Medicine, UCL
Professor Julian Gillmore, Department of Inflammation, Division of Medicine, UCL