IATP26d
Teen Dies Undergoing Experimental Gene Therapy
Jesse Gelsinger, a high school graduate who had suffered ... Gelsinger suffered from ornithine transcarbamylase (OTC) deficiency, a genetic disorder that affects mostly boys. The disease blocks the ...
BioWorld13d
Hope in OTC as Iecure’s in vivo gene editing trial shows first response
A 6.5-month-old boy with the rare inherited urea cycle disorder ornithine transcarbamylase (OTC) deficiency has responded positively in a targeted in vivo gene editing trial, in which a correct copy ...
Business Wire28d
Arcturus Therapeutics Announces Initiation of Dosing in Phase 2 Multiple Ascending Dose Studies for Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency
About Ornithine Transcarbamylase Deficiency Ornithine transcarbamylase (OTC) deficiency is the most common urea cycle disorder. Urea cycle disorders are a group of inherited metabolic disorders ...
Pharmaceutical Technology15d
ARCT-810 by Arcturus Therapeutics for Ornithine-Transcarbamylase Deficiency: Likelihood of Approval
ARCT-810 is under clinical development by Arcturus Therapeutics and currently in Phase II for Ornithine-Transcarbamylase Deficiency. According to GlobalData, Phase II drugs for ...
casereports.bmj19d
Undiagnosed partial ornithine transcarbamylase deficiency presenting as recurrent hyperammonaemic encephalopathy after capecitabine administration
Capecitabine is a widely used drug for cancer treatment. Capecitabine is a derivative of 5-fluorouracil (5-FU). A known complication of 5-FU is hyperammonaemia which can cause encephalopathy. To our ...