Conditions include chronic granulomatous disease and leukocyte adhesion deficiency syndrome ... and then delivered to the patient via an IV or subcutaneous injection weekly, biweekly, or monthly.

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 goal of the study is to alert medical professionals to clinical variability of PDHC deficiency and thus make easier early diagnosis. We present a spectrum of clinical phenotypes of six patients ...