N-acetylglutamate synthetase deficiency; OTC, ornithine transcarbamylase deficiency; PCD, pyruvate carboxylase deficiency; PA, propionic acidemia; THAN, transient hyperammonemia of the newborn ...
Hosted on MSN11mon
iECURE receives FDA IND clearance for OTC deficiency treatment trialpaving the way for a Phase I/II clinical trial of ECUR-506 to treat Ornithine Transcarbamylase (OTC) deficiency. Known as the OTC-HOPE study, the first-in-human trial will enrol newborn boys aged ...
Hosted on MSN2mon
Honoring a life taken too soon by giving back to othersJust days later doctors diagnosed him with a rare genetic disorder called Ornithine Transcarbamylase Deficiency (OTC). “I think the actual stats are about one in every 35,000 have a urea cycle ...
Arcturus has advanced the development of ARCT-810, an mRNA therapeutic candidate for ornithine transcarbamylase (OTC) deficiency. In December 2024, the Company initiated dosing of the first OTC ...
Reports Q4 revenue $22.8M, consensus $44.64M. “We continue to progress our flagship rare disease programs and look forward to sharing ...
Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF), along with its partnered mRNA vaccine programs ...
Arcturus Therapeutics Holdings Inc. (the "Company", "Arcturus", Nasdaq: ARCT), a commercial messenger RNA medicines company ...
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