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Pediatric Services In Netherlands
1 services found
by:Azafaros based inNaarden, NETHERLANDS
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 home-based, lifelong treatment, enhancing quality of life, particularly for pediatric patients, and reducing dependency on gene or enzyme replacement therapies. ...