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iECURE to Present Promising Initial Data from Gene Editing Trial for Neonatal OTC Deficiency
evaluating the gene-editing candidate ECUR-506 for neonatal onset ornithine transcarbamylase (OTC) deficiency, will be presented at the upcoming 2025 ACMG Annual Clinical Genetics Meeting in Los ...
Medscape10d
Not So Rare: Errors of Metabolism During the Neonatal Period
N-acetylglutamate synthetase deficiency; OTC, ornithine transcarbamylase deficiency; PCD, pyruvate carboxylase deficiency; PA, propionic acidemia; THAN, transient hyperammonemia of the newborn ...
Business Wire1mon
iECURE to Present Initial Data from Phase 1/2 OTC-HOPE Clinical Trial in Ornithine Transcarbamylase Deficiency at ACMG Annual Clinical Genetics Meeting
today announced that initial data from the Phase 1/2 OTC-HOPE clinical trial evaluating in vivo gene editing candidate ECUR-506 in neonatal onset ornithine transcarbamylase (OTC) deficiency will ...
American Association of Individual Investors7d
Why Arcturus Therapeutics Holdings Inc.’s (ARCT) Stock Is Up 6.84%
Arcturus Therapeutics Holdings Inc.’s ARCT share price has surged by 6.84%, which has investors questioning if this is right ...
Baby given new gene therapy in groundbreaking trial
It was thought that a transplant would be the only option for Tomas, until he was enrolled on to the groundbreaking gene therapy trial at Great Ormond Street Hospital (GOSH) in London. Symptoms of the ...
Nature3y
Arcturus—a clinical-stage mRNA therapeutics and vaccines company
self-amplifying mRNA vaccine for COVID-19 and an mRNA replacement therapy for ornithine transcarbamylase (OTC) deficiency (Fig. 1). Preclinical programs include wholly owned mRNA therapeutics for ...