Lysosomal storage disorders (LSDs) are a broad class of monogenic diseases with an
overall incidence of 1: 7,000 newborns, due to the defective activity of one or more …

Lysosomal storage diseases (LSDs) are a group of metabolic inborn errors caused by
defective enzymes in the lysosome, resulting in the accumulation of undegraded substrates …

Lysosomal Storage Diseases (LSDs) comprise a group of over fifty inherited metabolic
disorders, with their hallmark feature being deficient catabolism and accumulation (storage) …

Introduction: Lysosomal storage disorders (LSDs) encompass more than 50 distinct
diseases, caused by defects in various aspects of lysosomal function. Neurodegeneration …

Lysosomal storage disorders (LSDs) are a group of genetic diseases that result in metabolic
derangements of the lysosome. Most LSDs are due to the genetic absence of a single …

Introduction: Gene therapies can be envisioned for many disorders where conventional
therapies fall short. Lysosomal Storage Disorders (LSDs) are inherited, mostly monogenic …

The lysosomal storage disorders (LSD) are monogenic inborn errors of metabolism with
heterogeneous pathophysiology and clinical manifestations. In the last decades, these …

Lysosomal storage diseases (LSDs), as a group, are among the most common inherited
diseases affecting children. The primary defect is typically a genetic deficiency of one of the …

The lysosomal storage disorders (LSDs) comprise a heterogeneous group of inborn errors
of metabolism characterized by tissue substrate deposits, most often caused by a deficiency …

Lysosomal storage disorders (LSD) are a group of approximately 40 genetic diseases that
are caused by the deficiency of one or more lysosomal enzymes. The incidence of LSD is …