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Pediatric Patient Services Available In Canada Alberta

1 services found
Oral Treatment for Lysosomal Storage Disorders

Oral Treatment for Lysosomal Storage Disorders

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 administration allows for ...
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