Clinical Genomics Program Treatment Trials

Collaborations that uncovered the underlying genetic and cellular causes of specific immune disorders have, in some cases, resulted in novel treatments. Three such novel treatments are being actively investigated through a clinical treatment trial at NIH:

  1. Immunologic Effects of Supplemental Monosaccharide and Nucleoside Derivatives in Patients With Inherited Disorders of Glycosylation, 15-I-0159
  2. Study of Efficacy of CDZ173 in Patients With PASLI/APDS, NCT02435173
  3. Study of Magnesium Supplementation in Patients With XMEN Syndrome
  4. Phase 1/2 trial of amino acid supplementation in patients with atopic disease with or without a defect in the mTORC1 pathway


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