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 ...
OLPRUVA™ preliminary launch activities progressing; building out commercial and medical affairs teams to support U.S. launch in Q2 2023, with drug availability anticipated by early July 2023 Poster ...
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 ...
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 ...
A copy of the poster presentation from the 2023 SIMD Meeting is available on Acer’s website at: https://www.acertx.com/wp-content/uploads/2023/03/2023-SIMD-DCE ...
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 ...
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 ...
Scientists at Pacific Northwest Research Institute (PNRI) have overturned a long-held belief in genetics: that inheriting two harmful variants of the same gene always worsens disease. Instead, the ...
In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of ...
CAMP4 to Present New Preclinical Data from its Urea Cycle Disorders and SYNGAP1-Related Disorders Programs at the 28th American Society of Gene and Cell Therapy Annual Meeting Published Apr 28, 2025 4 ...
CELEBRATION, Fla. and ELK GROVE VILLAGE, Ill., June 18, 2024 (GLOBE NEWSWIRE) -- Orsini Specialty Pharmacy (Orsini), and Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra) today announced that Orsini ...
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