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The RAINBOW study: a phase 2 trial evaluating the 3-month pharmacokinetic and pharmacodynamic data and 18-month clinical and safety outcomes of nizubaglustat in GM2 gangliosidosis or Niemann–pick type C disease
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DOI：10.1016/j.ymgme.2026.110164.png)
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Abstract
En 中文
• Participants received nizubaglustat for up to 18 months in a phase 2 trial. • Nizubaglustat reduced plasma C16 and C18 glucosylceramides from baseline. • Seizure frequency decreased from baseline with nizubaglustat treatment. • Nizubaglustat was well tolerated, with no new or significant safety concerns. • Clinical outcomes of disease severity improved from baseline or remained stable.
Keywords:
nizubaglustat
GM2 gangliosidosis
Niemann–Pick type C disease
glucosylceramides
seizure frequency
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