Click Here to return To Results

 

Lentiviral Haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access Lancet 399:372-383, Fumagalli, F.,et al, 2022 See this aricle in Pubmed Article Abstract Treatment with arsa-cel resulted in sustained, clinically relevant benefits in children with early-onset MLD by preserving cognitive function and motor development in most patients and slowing demyelination and brain atrophy.

Related Tags

(click to filter results - removes previous filter) advances in neurology efficacy gene therapy metachromatic leukodystrophy safety stem cell transplantation treatment of neurologic disorder

Click Here to return To Results