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Safety and tolerability of losartan to treat recessive dystrophic epidermolysis bullosa in children (REFLECT): an open-label, single-arm, phase 1/2 trial.


ABSTRACT:

SUBMITTER: Kiritsi D 

PROVIDER: S-EPMC11558043 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature

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Safety and tolerability of losartan to treat recessive dystrophic epidermolysis bullosa in children (REFLECT): an open-label, single-arm, phase 1/2 trial.

Kiritsi Dimitra D   Schauer Franziska F   Gewert Stella S   Reineker Katja K   Reimer-Taschenbrecker Antonia A   Schwieger-Briel Agnes A   Ott Hagen H   Schmoor Claudia C   Grishina Olga O   Murrell Dedee D   Stiller Brigitte B   Zahn Tobias T   Nyström Alexander A   Bruckner-Tuderman Leena L  

EClinicalMedicine 20241030


<h4>Background</h4>Recessive dystrophic epidermolysis bullosa (RDEB) is a skin fragility disorder characterised by life-long mechanically induced skin blistering, fibrosis-driven pseudosyndactyly, and multi-organ involvement. Preclinical studies have suggested mitigated progression by angiotensin II type I receptor blockade through losartan. We aimed to determine the safety and tolerability of systemic losartan treatment among children with RDEB, and to obtain initial data on its clinical benefi  ...[more]

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