Migalastat

• CAT Number: I048027
• CAS Number: 108147-54-2
• Molecular Formula: C6H13NO4
• Molecular Weight: 163.2
• Purity: 98%
• Synonyms: 1-deoxygalactonojirimycin; 108147-54-2; (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol; DDIG
• Target: GLA
• Target Protein: LXBIFEVIBLOUGU-DPYQTVNSSA-N
• Appearance: Solid
• IC50: IC50: 0.04 μM (human α-Gal A)
• IUPAC Name: (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol
• InChI: InChI=1S/C6H13NO4/c8-2-3-5(10)6(11)4(9)1-7-3/h3-11H,1-2H2/t3-,4+,5+,6-/m1/s1
• SMILES: C1C(C(C(C(N1)CO)O)O)O

Migalastat is a medication used to treat Fabry disease, a rare genetic disorder. It works by stabilizing the mutated form of the alpha-galactosidase A enzyme, which is deficient in Fabry disease. This stabilization allows the enzyme to properly break down a fatty substance called globotriaosylceramide (Gb3), which accumulates in the body and causes the symptoms of Fabry disease. Migalastat is specifically used to treat Fabry disease in patients who have a certain genetic mutation, known as amenable mutations. It is taken orally and has been shown to reduce Gb3 levels in the body, improving symptoms and slowing disease progression in some patients.