In collaboration with Great Ormond Street Hospital and Children's Charity (GOSH) and the NIHR we are so pleased that Mission EB has started, the first large-scale stem cell clinical trial for children with EB. Researchers from GOSH have begun the largest ever stem cell clinical trial for...
The first global congress on Epidermolysis Bullosa took place on 19-23 January, in London. ...
Krystal Biotech have announced that the first patient has been dosed in the Phase 1/2 clinical trial of its drug, KB103, for the treatment of dystrophic epidermolysis bullosa (DEB). The Phase 1/2 trial at Stanford University is a single-centre, open-label, placebo-controlled study conducting an intra-subject comparison...
In 2016, together with two other US based medical research charities, we funded the EB iPS Cell Consortium, which includes researchers from leading universities in the US. The results of its early work were so promising that it leveraged a major grant of $3.8 million,...
Sharmila Nikapota, the mother of a child with a rare genetic disorder, has high hopes for gene editing...