Washington Examiner

Urea-cycle disorder treatment reviewed

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 ...
Business Insider

NUCDF and Zevra Therapeutics Launch Check Ammonia Campaign to Highlight Critical Importance of Prompt, Accurate Blood Ammonia Testing for Diagnosing Urea Cycle Disorders

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 ...

Ultragenyx posts late-stage trial win for gene therapy in urea cycle disorder

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 ...
Monthly Prescribing Reference

Olpruva Approved for Patients With Urea Cycle Disorders

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 ...
EurekAlert!

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 ...
Nature

Arginase Deficiency and Urea Cycle Disorders

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 ...
FiercePharma

Zevra, nonprofit team up to urge timely testing for life-threatening rare diseases

Zevra Therapeutics has partnered with the National Urea Cycle Disorders Foundation (NUCDF) to run an awareness campaign designed to improve the diagnosis of conditions treated by one of its products.
Nasdaq

CAMP4 Therapeutics Reports Positive Results in SYNGAP1-Related Disorders and Urea Cycle Disorders at ASGCT Annual Meeting

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 ...
Seeking Alpha

Acer Therapeutics Announces Data Presented at Society for Inherited Metabolic Disorders Annual Meeting Identifying Preferred Urea Cycle Disorder Treatment Attributes

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 ...
Nasdaq

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

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 ...
Seeking Alpha

Zevra Therapeutics Transitions to Orsini as the Specialty Pharmacy Provider for OLPRUVA® (sodium phenylbutyrate), a Treatment for Certain Urea Cycle Disorders

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 ...