Mesenchymal intravenous stromal cell infusions in children with recessive dystrophic epidermolysis bullosa: MissionEB protocol for a randomised, double-blinded, placebo-controlled, two-centre, crossover trial with an internal phase I dose de-escalation phase and open-label extension

Mesenchymal intravenous stromal cell infusions in children with recessive dystrophic epidermolysis bullosa: MissionEB protocol for a randomised, double-blinded, placebo-controlled, two-centre, crossover trial with an internal phase I dose de-escalation phase and open-label extension

Introduction Recessive dystrophic epidermolysis bullosa (RDEB) is a severe genetic mucocutaneous fragility disorder characterised by chronic blistering, slow wound healing and increased risk of squamous cell carcinoma. Current management options are very limited.Methods This is a randomised (1:1), p...

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Main Authors: Cindy Cooper, Katie Biggs, Munyaradzi Dimairo, Steven A Julious, Gabriela Petrof, John A McGrath, Diana Papaioannou, Anna E Martinez, Paul Tappenden, Marie-Louise Lovgren, Malobi Ogboli, Rachel Glover, Maria L Bageta, Pablo López-Balboa, Kate Hutchence, Katherine Ennis, Pratima Poudel, Muna Nadeem
Format: Article
Language:English
Published: BMJ Publishing Group 2025-05-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/15/5/e089857.full
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https://bmjopen.bmj.com/content/15/5/e089857.full

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