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 ...

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 ...

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 ...

Ultragenyx Pharma (RARE) stock is in focus as gene therapy DTX301 met its main goal in a late-stage trial for the most common urea cycle disorder. Read more here.

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 ...

CAMP4 Therapeutics reports promising results for CMP-SYNGAP-01 and CMP-CPS-001 in treating genetic disorders, with favorable safety data. CAMP4 Therapeutics announced promising results from its ...

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 ...

DUBLIN, Ireland, March 22, 2017 (GLOBE NEWSWIRE) -- Horizon Pharma plc (NASDAQ:HZNP), a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring and ...

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 / ...

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 ...