A single course of IV gentamicin may be a safe and readily available systemic therapy for patients with junctional epidermolysis bullosa, according to results from a small pilot trial.

While the long-term safety and efficacy remains unknown, US researchers say their findings provide a glimmer of hope for those born with the incurable blistering skin disorder.

In a previous study, the University of Southern California-led team had shown that topical gentamicin generated new laminin 332 — which is essential for epithelial cell adhesion to the basement membrane — and enhanced skin wound healing in affected patients.

In the current study, published in JAMA Dermatology, they gave a course of IV gentamicin readthrough therapy to five paediatric patients aged three months to 10 years with junctional epidermolysis bullosa (JEB).