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Lysosomal Storage Disease Services
2 services found
by:Azafaros based inNaarden, NETHERLANDS
Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These disorders are individually rare but collectively affect 1 in 5,000 live births. ...
by:Azafaros based inNaarden, NETHERLANDS
Their lead compound, nizubaglustat, leverages unique dual-mode action, making it a promising candidate for modifying diseases irrespective of patient genotypes. Notably effective against conditions such as GM1 and GM2 gangliosidoses and Niemann-Pick disease type C, nizubaglustat has shown commendable safety and tolerability in initial human studies. Oral ...