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 ...

Ultragenyx Pharmaceutical (RARE) announced on Thursday that its gene therapy candidate DTX301 reached the main goal in a late-stage trial for ornithine transcarbamylase (OTC) deficiency, the most ...

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

Nominated KRRO-121 development candidate for the potential treatment of hyperammonemia in patients with urea cycle disorders and hepatic ...

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 ...

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. (ZVRA) (Zevra) today announced that Orsini is now the ...