Indication
Lysosomal Storage Disease
2 clinical trials
1 product
Clinical trial
A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic LeukodystrophyStatus: Active (not recruiting), Estimated PCD: 2018-04-09
Product
OTL-200Clinical trial
A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)Status: Active (not recruiting), Estimated PCD: 2022-04-06