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A single patient trial with Ataluren in one case of severe common variable immunodeficiency with autoimmunity due to homozygous stop codon mutations of LRBA - LRBA01

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Trial Profile

A single patient trial with Ataluren in one case of severe common variable immunodeficiency with autoimmunity due to homozygous stop codon mutations of LRBA - LRBA01

Status: Discontinued
Phase of Trial: Phase II

Latest Information Update: 09 Sep 2025

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At a glance

  • Drugs Ataluren (Primary)
  • Indications Immunological disorders
  • Focus Therapeutic Use

Most Recent Events

  • 30 Sep 2035 The term LRBA deficiency (called LATAIE disease) is not present in the MeSH. Hence i indexed immunological disorders as of now. Please update further.
  • 09 Sep 2025 New trial record

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