Advances in therapies for neurological Lysosomal Storage Disorders.
Sign Up to like & getrecommendations! Published in 2023 at "Journal of inherited metabolic disease"
DOI: 10.1002/jimd.12615
Abstract: Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic diseases caused by functional defects in specific lysosomal proteins. The lysosome is a cellular organelle that plays a critical role in catabolism of waste… read more here.
Keywords: therapies neurological; storage disorders; neurological lysosomal; lysosomal storage ... See more keywords
[Antisense therapies for neurological diseases].
Sign Up to like & getrecommendations! Published in 2019 at "Der Nervenarzt"
DOI: 10.1007/s00115-019-0724-4
Abstract: Despite identification of many genes causing neurodegenerative diseases in the last decades, development of disease-modifying treatments has been slow. Antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy, Duchenne muscular dystrophy and transthyretin amyloidosis predict a robust… read more here.
Keywords: medicine; neurological diseases; antisense therapies; therapies neurological ... See more keywords