来自MSN2 个月
Urea cycle disorder treatment market to hit $215.4m by 2028
The global urea cycle disorder (UCD ... for advanced screening and UCD management is growing due to a rise in metabolic disorders. Despite the market’s growth, challenges remain due to limited ...
Medscape4 天
Not So Rare: Errors of Metabolism During the Neonatal Period
Steiner, MD Neonates with profound and prolonged hyperammonemia with coma due to urea cycle defect will have ... Division of Metabolism, Departments of Pediatrics and Molecular and Medical ...
Nature4 年
Synlogic probiotics turn up the heat on cancer and metabolic disease
The company pipeline includes programs targeting metabolic diseases ... targets hyperammonemia in individuals born with urea cycle disorders (UCDs). It has been given both Orphan Drug and Fast ...
BioWorld25 天
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 ...