RyR1 Structural Alterations Explain Statin-Associated Muscle Dysfunction
December 16, 2025
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Brand Name :
N/A
Synonyms :
nizubaglustat
Class :
GBA2 protein inhibitors, Glucosylceramide synthase inhibitors
Brand Name :
N/A
Synonyms :
nizubaglustat
Class :
GBA2 protein inhibitors, Glucosylceramide synthase inhibitors
Adult
Dosage Forms & Strengths
It is used as an investigational and research purpose
Tablet
1 mg
3 mg
9 mg
It is under development for the treatment of subjects with neurological manifestations in primary and secondary gangliosidoses
In the treatments for lysosomal storage disorders with neurological implications
Not determined
Actions and Spectrum:
Nizubaglustat is an inhibitor of ceramide glucosyltransferase. It also inhibits GBA2, involving neuropathological lysosomal storage disorders.
Adverse reaction
None
Black box warning
None
Contraindication/Caution:
Contraindications
Hypersensitivity
Cautions
Renal impairment
Hepatic impairment
Pregnancy
Pregnancy Warnings:
Pregnancy category: N/A
Lactation: Excretion of the drug into the human breast milk is unknown
Pregnancy categories:
Pharmacology:
Nizubaglustat is a novel azasugar-based pharmacological agent featuring a nitrogen-containing ring. It will effectively cross the blood-brain barrier.
Pharmacodynamics:
Nizubaglustat is a novel oral drug targeting ceramide glucosyltransferase and non-lysosomal neutral glucosylceramidase. The drug effectively reduced key biomarkers in plasma and cerebrospinal fluid.
Pharmacokinetics:
Limited information is available on ADME.
Administration:
It is taken orally.
Patient information leaflet
Generic Name: nizubaglustat
Why do we use nizubaglustat?
It is under clinical study to find treatments for lysosomal storage disorders along with primary and secondary gangliosidoses.
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