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 ...

New FDA Plausible Mechanism Framework Spurs Development of Personalized In Vivo Prime Editing Platform for Urea Cycle Disorders

Guidance opens doors for rare disease treatments, but academic teams will likely need industry partners to meet rigor required for FDA approval PHILADELPHIA, March 31, 2026 /PRNewswire/ -- Researchers ...
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 ...
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 ...
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

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 ...
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.
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 ...
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 ...
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 ...
Business Insider

Horizon Pharma plc to Present Data on RAVICTI® (glycerol phenylbutyrate) Oral Liquid for Urea Cycle Disorder Patients Aged Two Months to Two Years

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 ...