Interest in lysosomal storage diseases (LSDs) has increased significantly, because several new therapeutics received FDA approval in 2003. Though long overlooked by most major pharmaceutical ...
Treatment of neuronopathic lysosomal storage diseases (LSD) represents an exceptional challenge for the research community, as a substance does not only have to be active in the periphery but needs ...
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Passage Bio Out-Licenses Rare Disease Programs to GEMMA Biotherapeutics“We are excited to have out-licensed our pediatric lysosomal storage disease programs to a company deeply committed to ...
doctors.org.uk Lysosomal storage disorders (LSD) comprise a group of more than 40 diseases caused by a deficiency of lysosomal enzymes, membrane transporters or other proteins involved in lysosomal ...
It can also be used to improve outcomes of children with lysosomal storage diseases such as cystinosis by early detection and intervention. Dr. Hahn’s method uses dried blood spots (DBS) on filter ...
Lysosomal acid lipase (LAL) deficiency is a rare autosomal recessive disorder which causes two distinct clinical phenotypes: Wolman's disease and cholesterol ester storage disease. LAL hydrolyses ...
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