Introduction Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …

The ability to learn language is a human trait. In adults and children, brain imaging studies
have shown that auditory language activates a bilateral frontotemporal network with a left …

In adults, specific neural systems with right-hemispheric weighting are necessary to process
pitch, melody, and harmony as well as structure and meaning emerging from musical …

Background Metachromatic leukodystrophy (a deficiency of arylsulfatase A [ARSA]) is a fatal
demyelinating lysosomal disease with no approved treatment. We aimed to assess the long-…

Background Effective treatment for metachromatic leukodystrophy (MLD) remains a substantial
unmet medical need. In this study we investigated the safety and efficacy of atidarsagene …

Background Allogeneic hematopoietic stem-cell transplantation is the standard of care for
Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this …

BACKGROUND AND PURPOSE: Diffusion-weighted MR imaging studies of normal brain
development have focused on premature babies who were free of focal lesions on …

BACKGROUND AND PURPOSE: MR imaging is the method of choice for assessment in
vivo of the development of myelination of the human central nervous system. During the first …

Background Etiopathogenesis of preterm birth (PTB) is multifactorial, with a universe of risk
factors interplaying between the mother and the environment. It is of utmost importance to …

In this study, we characterize the natural course of metachromatic leukodystrophy (MLD),
explore intra/inter group differences, and identify biomarkers to monitor disease progression. …