A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher
Both coronary plaque burden and the rate of plaque progression are increased in patients with high Lp(a) levels, potentially explaining the link with higher CV risk, new findings suggest.
Form 424B3 NewAmsterdam Pharma Co streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.
16.02.2023 - - Rapid, significant, and sustained increase in plasma pyrophosphate (PPi) observed and encouraging patient reported outcome data in all dose cohorts in ENPP1 Deficiency trial - - Rapid and significant increase in PPi observed in all dose cohorts .