VS-01 for the treatment of Urea Cycle Disorder (UCD) and Organic Acidemia Disorder (OAD); GNS561, which is in Phase 1b/2a trial to treat patients with cholangiocarcinoma (CCA); VS-01-ACLF and ...

Urine organic acid testing confirmed the presence of orotic aciduria along with homozygous variations in the UMPS gene ... A referral of any positive NBS for a metabolic disorder includes the option ...

CSPD, carbamyl phosphate synthetase deficiency; HHH, hyperornithinemia hyperammonemia homocitrullinuria syndrome; IVA, isovalemic acidemia; LCHADD, long chain 3-chydroxy-acyl-CoA dehydrogenase ...

ACLF clinical trials expected to initiate in 1Q253 clinical data readouts expected by year-end 2025 across the ACLF pipeline, including ...

Correspondence to Dr Cecilia Marelli, Department of Neurology, Expert Center for Neurogenetic Diseases and Adult Mitochondrial and Metabolic Diseases, Gui de Chauliac University Hospital, Montpellier, ...

ACLF clinical trials expected to initiate in 1Q253 clinical data readouts expected by year-end 2025 across the ACLF pipeline, including on-going UNVEIL-IT® Phase 2 trialData of on-going Phase 1b ...

Feb. 5, 2025 — New research has shown that diet could influence the risk of both depression and Alzheimer's disease (AD). Evidence suggests that depressive symptoms are both a risk factor for AD ...

Neurodegenerative diseases are a heterogeneous group of disorders that are characterized by the progressive degeneration of the structure and function of the central nervous system or peripheral ...