Plasma Progerin in Patients With Hutchinson-Gilford Progeria Syndrome: Immunoassay Development and Clinical Evaluation

CONCLUSIONS: A sensitive, quantitative immunoassay for progerin was developed and used to demonstrate high progerin levels in HGPS plasma that decreased with lonafarnib therapy. The extent of improved survival was associated with both the magnitude of progerin decrease and duration at lower levels. Thus, plasma progerin is a biomarker for HGPS whose reduction enables short- and long-term assessment of progerin-targeted treatment efficacy.REGISTRATION: URL: https://www.CLINICALTRIALS: gov. Unique identifiers: NCT00879034 and NCT00916747.PMID:36919608 | DOI:10.1161/CIRCULATIONAHA.122.060002

Source: Atherosclerosis - March 15, 2023 Category: Cardiology Authors: Leslie B Gordon Wendy Norris Sarah Hamren Robert Goodson Jessica LeClair Joseph Massaro Asya Lyass Ralph B D'Agostino Kelsey Tuminelli Mark W Kieran Monica E Kleinman Source Type: research