Many neurodegenerative diseases are characterized by the accumulation of undegradable molecules in cells or at extracellular sites in the brain. One such family of diseases is the lysosomal storage disorders, which result from defects in various aspects of lysosomal function. Until recently, there was little prospect of treating storage diseases involving the CNS. However, recent progress has been made in understanding these conditions and in translating the findings into experimental therapies. We review the developments in this field and discuss the similarities in pathological features between these diseases and some more common neurodegenerative disorders
Lysosomal storage disorders (LSDs) constitute a large group of rare, multisystemic, progressive, inh...
Lysosomal storage diseases (LSDs) include approximately 70 distinct disorders that collectively acco...
Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosoma...
Pharmacological research has always focused on developing new therapeutic strategies capable of modi...
Lysosomal storage disorders are inherited metabolic diseases in which a mutation in a gene encoding ...
There are over 40 human disease states that are caused by defects in various aspects of lysosomal fu...
AbstractThere are over 40 human disease states that are caused by defects in various aspects of lyso...
Lysosomal storage disorders (LSDs) are rare diseases caused by inherited mutations in genes coding f...
Lysosomal storage diseases are a group of rare, inborn, metabolic errors characterized by deficienci...
Although neurodegenerative diseases are most prevalent in the elderly, in rare cases, they can also ...
Over the past three decades the lysosomal storage diseases have served as model for rare disease tre...
Abstract In the last decades, it has become more and more evident that lysosomal storage disorders a...
The blood-brain barrier becomes a crucial issue in neuronopathic lysosomal storage diseases for thre...
Although the first description of a lysosomal storage disorder was that of Tay-Sachs disease in 1881...
International audienceRare genetic diseases affect a limited number of patients, but their etiology ...
Lysosomal storage disorders (LSDs) constitute a large group of rare, multisystemic, progressive, inh...
Lysosomal storage diseases (LSDs) include approximately 70 distinct disorders that collectively acco...
Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosoma...
Pharmacological research has always focused on developing new therapeutic strategies capable of modi...
Lysosomal storage disorders are inherited metabolic diseases in which a mutation in a gene encoding ...
There are over 40 human disease states that are caused by defects in various aspects of lysosomal fu...
AbstractThere are over 40 human disease states that are caused by defects in various aspects of lyso...
Lysosomal storage disorders (LSDs) are rare diseases caused by inherited mutations in genes coding f...
Lysosomal storage diseases are a group of rare, inborn, metabolic errors characterized by deficienci...
Although neurodegenerative diseases are most prevalent in the elderly, in rare cases, they can also ...
Over the past three decades the lysosomal storage diseases have served as model for rare disease tre...
Abstract In the last decades, it has become more and more evident that lysosomal storage disorders a...
The blood-brain barrier becomes a crucial issue in neuronopathic lysosomal storage diseases for thre...
Although the first description of a lysosomal storage disorder was that of Tay-Sachs disease in 1881...
International audienceRare genetic diseases affect a limited number of patients, but their etiology ...
Lysosomal storage disorders (LSDs) constitute a large group of rare, multisystemic, progressive, inh...
Lysosomal storage diseases (LSDs) include approximately 70 distinct disorders that collectively acco...
Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosoma...