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Brand Name :
N/A
Synonyms :
arimoclomol
Class :
Pharmacologic Chaperones
Brand Name :
N/A
Synonyms :
arimoclomol
Class :
Pharmacologic Chaperones
FDA Approval Pending for treatment of Niemann-Pick disease Type C
Not suggested
Refer to adult dosing
Actions and Spectrum
arimoclomol is an inducer of heat shock proteins (HSPs). Heat shock proteins are a group of cellular chaperones that help maintain proper protein folding and prevent the accumulation of misfolded or aggregated proteins.
None
Black Box Warning:
None
Contraindication/Caution:
None
Pregnancy warnings:
Pregnancy category: N/A
Lactation: Excretion into human milk is unknown
Pregnancy Categories:
Category A: well-controlled and Satisfactory studies show no risk to the fetus in the first or later trimester.
Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women.
Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.
Category D: adequate data with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.
Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.
Category N: There is no data available for the drug under this category.
Pharmacology
arimoclomol increases the synthesis of heat shock proteins (HSPs), which protect cells from damage, but only in cells that are physiologically stressed, such as those affected by illness.
In order to repair cells and lessen protein misfolding and aggregation, it activates proteins that serve as pharmacologic chaperones. This enables the cells to handle accumulated lipid.
Pharmacodynamics
Patients with Niemann-Pick disease (NPD) type C disease are unable to transfer cholesterol and lipids within cells, resulting in an abnormal accumulation of these substances within a variety of bodily tissues, including the brain tissue.
Pharmacokinetics
Limited information available on ADME
Administration
arimoclomol is administered orally in the form of tablets or capsules.
Patient information leaflet
Generic Name: arimoclomol (FDA Approval Pending)
Why do we use arimoclomol?
arimoclomol has been investigated in clinical trials for the treatment of Niemann-Pick disease Type C (NPD), a rare and progressive genetic disorder characterized by impaired lipid metabolism.
arimoclomol has undergone clinical trials as a potential treatment for Amyotrophic Lateral Sclerosis (ALS). It is a progressive neurodegenerative disease affecting motor neurons.
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