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These mice exhibit a severe bleeding tendency and a leukocyte adhesion defect reminiscent to the phenotype in LAD-III patients. Kindlin-3 belongs to a family of three cytoplasmic proteins ...
Patients in the EU with Fanconi anaemia ... from the FDA on Kresladi (marnetegragene autotemcel) for severe leukocyte adhesion deficiency-I (LAD-I), its lead product, which is due by 30th June ...
The company is developing its gene therapy candidate, Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder.