2 days ago
Breakthrough for children with rare, painful skin condition
Gabrielius Misurenkovas, a 12-year-old boy with the rare, painful skin condition, recessive dystrophic epidermolysis bullosa (RDEB), has gained more freedom thanks to a new clinical trial.
RDEB causes extremely fragile skin, leading to blistering, deep wounds, and scarring, with only around 150 children in the UK affected and no prior specific treatment available.
Gabrielius participated in a clinical trial at Great Ormond Street Hospital and Birmingham Children's Hospital, receiving intravenous infusions of mesenchymal stromal cells (MSC), specifically CORDStrom.
The trial significantly improved his condition, leading to quicker wound healing, reduced inflammation and itchiness, and allowing him to participate in activities like playing football and cycling.
The company INmuneBio is providing CORDStrom free for a year to trial participants and is seeking regulatory approval for the treatment in the UK and other countries.
