PASADENA, Calif. and CELEBRATION, Fla., Oct. 09, 2024 (GLOBE NEWSWIRE) -- The National Urea Cycle Disorders Foundation (NUCDF) and Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) are teaming up to launch an ...
Nitrogen is a big part of the air we breathe, but too much can kill. Researchers at Johns Hopkins University reviewed 25 years of studies on using sodium phenylacetate and sodium benzoate to help ...
Arginase deficiency is a rare autosomal recessive metabolic disorder that disrupts the final step of the urea cycle, impairing the conversion of arginine into urea and ornithine. In affected ...
The application was approved through the FDA’s 505(b)(2) regulatory pathway. The Food and Drug Administration (FDA) has approved Olpruva ™ (sodium phenylbutyrate) as an oral suspension for the ...
National UCD Foundation to build network, create roadmap for future research in urea cycle disorders
The National Urea Cycle Disorders Foundation (NUCDF), the leading patient advocacy group for people affected by urea cycle disorders (UCDs), today announced the launch of a two-year national project ...
The European Commission (EC) granted orphan designation to iECURE's lead product GTP-506 to treat Ornithine Transcarbamylase (OTC) deficiency. OTC deficiency is a type of urea cycle disorder. It is a ...
Preclinical data to showcase the potential of regRNA-targeting to increase protein levels in a clinically meaningful way by upregulating gene expression Interim safety data including patient ...
Acer Therapeutics reports data from a survey of UCD healthcare providers show taste and odor are the most important attributes when considering treatment options and adherence GENEVA, SWITZERLAND / ...
Researchers found that paired pathogenic variants can restore protein function rather than make it worse. This phenomenon ...
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