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CAS 75172-81-5

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Migalastat hydrochloride

Description:
Migalastat hydrochloride is a pharmacological agent primarily used in the treatment of Fabry disease, a genetic disorder resulting from the deficiency of the enzyme alpha-galactosidase A. This compound acts as a chaperone, stabilizing the enzyme and facilitating its proper function, which helps to reduce the accumulation of certain lipids in the body. Chemically, Migalastat hydrochloride is characterized by its specific molecular structure, which includes a chiral center, contributing to its biological activity. It is typically administered orally and is known for its favorable pharmacokinetic profile, including good absorption and distribution in the body. The hydrochloride salt form enhances its solubility and stability. Migalastat hydrochloride has undergone extensive clinical trials, demonstrating its efficacy in reducing disease-related symptoms and improving quality of life for patients with Fabry disease. As with any medication, it is essential to consider potential side effects and contraindications, and it should be used under the guidance of a healthcare professional.
Formula:C6H13NO4·ClH
InChI:InChI=1S/C6H13NO4.ClH/c8-2-3-5(10)6(11)4(9)1-7-3;/h3-11H,1-2H2;1H/t3-,4+,5+,6-;/m1./s1
InChI key:InChIKey=ZJIHMALTJRDNQI-OLALXQGDSA-N
SMILES:C(O)[C@@H]1[C@H](O)[C@H](O)[C@@H](O)CN1.Cl
Synonyms:
  • 1-Deoxygalactonojirimycin hydrochloride
  • 3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride (1:1), (2R,3S,4R,5S)-
  • 3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride, (2R,3S,4R,5S)-
  • 3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride, [2R-(2α,3α,4α,5β)]-
  • Galafold
  • Migalastat HCl
  • Migalastat hydrochloride
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Found 13 products.