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Metachromatic Leukodystrophy (MLD) research hub
3 indexed studies · 0 currently recruiting
- active not recruitingPHASE2
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
This study is looking at a new medicine called SHP611 for children with Metachromatic Leukodystrophy (MLD). Doctors want to see if SHP611, given into the spinal fluid, can help children keep their ability to move around for longer. It also looks at how it affects speech and if it's safe.
United States · Argentina · Belgium - ongoing, recruitingTherapeutic confirmatory (Phase III)
A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA, for the treatment of early onset Metachromatic Leukodystrophy (MLD)
Italy - ongoing, recruitingPhase I and Phase II (Integrated)- Other
A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy
Italy