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ISA 2024 | Choosing between standard of care and clinical trial enrollment in patients with amyloidosis
Chafic Karam, MD, University of Pennsylvania, Philadelphia, PA, discusses which patients with amyloidosis should be treated with standard-of-care regimens and which may be better suited to a clinical trial. For patients with neuropathy, current drugs, especially silencers, are very effective when started early. Most clinical trials focus on cardiomyopathy, often enrolling patients with advanced heart failure to evaluate different therapies. Dr Karam highlights that future clinical trials should investigate pharmaceutical prevention strategies for gene carriers who have not yet developed disease symptoms. This interview took place at the XIX International Symposium on Amyloidosis (ISA) in Rochester, MN.
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