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Late Metachromatic Leukodystrophy (MLD) research hub
2 indexed studies · 0 currently recruiting
- ongoing, recruitingTherapeutic exploratory (Phase II)
A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy
This study is looking into a new treatment called rhASA for Late Infantile Metachromatic Leukodystrophy (MLD), a serious inherited illness. It's testing if rhASA can help children keep their ability to move and whether it can slow down the disease's progression.
Germany · France · Belgium - ongoing, recruitingPhase I and Phase II (Integrated)- Other
An Open-Label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy
This study is looking at a new treatment called rhASA for a rare genetic condition in children and young people called Metachromatic Leukodystrophy (MLD). It's checking if the treatment is safe and if it helps with MLD symptoms over a long time.
Czechia · Denmark · Germany