A courageous 12-year-old boy endures unimaginable pain due to a rare genetic disorder causing his skin to tear at the slightest touch. His family shares their heartbreaking journey and hopes for better treatments.
A groundbreaking gene therapy trial at Great Ormond Street Hospital is transforming lives for children with recessive dystrophic epidermolysis bullosa, offering new hope for a cure.
A British toddler with a devastating genetic skin condition has seen remarkable improvements after receiving pioneering gene therapy treatment, offering hope to families affected by the rare disease.