Congratulations to Eric Mallack, M.D. and Weill Cornell Medicine collaborators on the approval of their upcoming international gene therapy trial entitled, “A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy)." Dr. Eric Mallack will serve as Primary Investigator of the study.
Infantile Krabbe disease, or globoid cell leukodystrophy, is a genetic disorder of the nervous system that usually results in death before the age of two. It is caused by mutations in the GALC gene which encodes an enzyme responsible for the breakdown of multiple lipids in the brain. In Krabbe disease, there is a toxic accumulation of these lipids in cells throughout the nervous system. This results in progressive neurodegeneration: brain atrophy, spasticity, loss of hearing and vision, seizures, weight loss, aspiration, loss of development milestones and early mortality.
