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ey0018.3-12 | Clinical studies | ESPEYB18

3.12. Randomised trial of block and replace vs. dose titration thionamide in young people with thyrotoxicosis

CL Wood , M Cole , M Donaldson , DB Dunger , R Wood , N Morrison , JNS Matthews , SHS Pearce , TD Cheetham

Eur J Endocrinol. 2020;183:637645. doi: 10.1530/EJE-20-0617.This study provides for the first time clear evidence that the block and replace (BR) strategy (combination of carbimazole plus levothyroxine) is not superior to the carbimazole dose titration (DT) strategy for treatment of paediatric Graves disease.Wood et al. present the first randomized c...

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ey0019.14-13 | New treatments | ESPEYB19

14.13. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis

Gillmore Julian D , Gane Ed , Taubel Jorg , Kao Justin , Fontana Marianna , Maitland Michael L , Seitzer Jessica , O'Connell Daniel , Walsh Kathryn R , Wood Kristy , Phillips Jonathan , Xu Yuanxin , Amaral Adam , Boyd Adam P , Cehelsky Jeffrey E , McKee Mark D , Schiermeier Andrew , Harari Olivier , Murphy Andrew , Kyratsous Christos A , Zambrowicz Brian , Soltys Randy , Gutstein David E , Leonard John , Sepp-Lorenzino Laura , Lebwohl David

N Engl J Med. 2021 Aug 5;385(6):493502. PMID: 34215024 doi: 10.1056/NEJMoa2107454. Epub 2021 Jun 26.Brief Summary: 6 patients with hereditary, life-threatening transthyretin amyloidosis were treated with a novel in vivo gene-editing therapeutic agent (NTLA-2001) based on CRISPR-Cas9 technology. The new agent was given intravenously in a single dose and was able to durably knockou...

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ESPE Yearbook of Paediatric Endocrinology

3.12. Randomised trial of block and replace vs. dose titration thionamide in young people with thyrotoxicosis

14.13. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis

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