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Arimoclomol

Structure via PubChem · Public domain (PubChem)

EntityQ4790694· pop 5· linked from 28 articles

Arimoclomol

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Arimoclomol, sold under the brand name Miplyffa, is a medication used for the treatment of Niemann–Pick disease type C. It is taken by mouth.

Chemical data

Formula
C14H20ClN3O3
Molecular weight
313.78 g/mol
IUPAC name
N-[(2R)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl chloride
SMILES
C1CCN(CC1)C[C@H](CON=C(C2=C[N+](=CC=C2)[O-])Cl)O
InChIKey
SGEIEGAXKLMUIZ-CYBMUJFWSA-N
XLogP
1.2
Polar surface area
70.5 Ų
H-bond donors
1
H-bond acceptors
5
Formal charge
0

via PubChem

Drug data · ChEMBL

Max clinical phase
Approved
Molecule type
Small molecule
First approval
2024
Admin. routes
oral
Indications
inclusion body myositis, amyotrophic lateral sclerosis, Gaucher disease, Niemann-Pick disease type C

via ChEMBL · EBI

Wikidata facts

Mass
313.11931918
Show 4 more facts
chemical formula
C₁₄H₂₀ClN₃O₃
canonical SMILES
C1CCN(CC1)CC(CON=C(C2=C[N+](=CC=C2)[O-])Cl)O
isomeric SMILES
c1cc(c[n+](c1)[O-])C(=NOC[C@@H](CN2CCCCC2)O)Cl
Commons category
Arimoclomol
Sources (2)

via Wikidata · CC0

~4 min read

Encyclopedic overview

9 sections
Contents
  • Medical uses
  • Side effects
  • Mechanism of action
  • History
  • Society and culture
  • Legal status
  • Names
  • Research
  • References

Arimoclomol, sold under the brand name Miplyffa, is a medication used for the treatment of Niemann–Pick disease type C. It is taken by mouth.

The most common side effects include upper respiratory tract infection, diarrhea, and decreased weight.

Excerpted from Wikipedia’s “Arimoclomol” article, available under the CC BY-SA 4.0 licence.

Available in 4 languages

via Wikidata sitelinks · CC0