Clinical Trial: Expanding the Spectrum of Adenosine Deaminase 2 (ADA2) Deficiency: Towards a Gene Therapy Approach

U.S., Oct. 21 -- ClinicalTrials.gov registry received information related to the study (NCT07882680) titled 'Expanding the Spectrum of Adenosine Deaminase 2 (ADA2) Deficiency: Towards a Gene Therapy Approach' on Oct. 12.

Brief Summary: Adenosine deaminase 2 deficiency (DADA2), caused by autosomal recessive mutations in the ADA2 gene (OMIM: 607575), is an autoinflammatory disease characterized by early-onset inflammatory vasculopathy, stroke, and immunodeficiency (cytopenias and antibody deficiency). Due to its wide clinical variability and heterogeneous presentation, a large proportion of patients with DADA2 remain undiagnosed.

Anti-TNF therapy is effective in DADA2 patients with inflammatory vasculopathy; however, its long-term efficacy ...