Article
New therapeutic approach by sirolimus for enteropathy treatment in patients with LRBA deficiency.
European annals of allergy and clinical immunology - 1 Sept 2017
Azizi G, Abolhassani H, Yazdani R, Mohammadikhajehdehi S, Parvaneh N, Negahdari B, Mohammadi J, Aghamohammadi A
Abstract excerpt
SUMMARY: Purpose. To report the successful use of sirolimus for management of enteropathy in four patients with LPS-responsive beige-like anchor protein (LRBA) deficiency. Methods. Case series. Results. sirolimus therapy led to a complete improvement of symptoms including decrease in frequency and severity of diarrhea, as well as patients' weight gain. No signs of abdominal cramps and anorexia were also detected...
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