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Single-patient Ataluren trial in LRBA deficiency with severe common variable immunodeficiency and autoimmunity
2024-518919-19-00Phase 2Completed
- Sponsor
- Centre Hospitalier Universitaire de Liège
- Start
- Nov 2024
- Modality
- Small Molecules
- Countries
- 1
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Overview
Phase 2, open-label, single-arm trial of ataluren in LRBA deficiency (LRBA), common variable immunodeficiency (CVID) and autoimmune disease, sponsored by Centre Hospitalier Universitaire de Liège. Started Nov 2024; status: completed.
02
Drugs
ataluren
03
Conditions
LRBA deficiency (LRBA)common variable immunodeficiency (CVID)autoimmune disease
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