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75172-81-5

3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride,[2R-(2a,3a,4a,5b)]-

Catalog#: AR01CC3P  |   CAS#: 75172-81-5  |   MDL#: MFCD00269962  |   MF: C6H14ClNO4  |   MW: 199.6327

Packsize Purity Price Availability Quantity
1mg 98% $30.00 Global Stock Buy Now Add To Cart
5mg 98% $90.00 Global Stock Buy Now Add To Cart
10mg 98% $135.00 Global Stock Buy Now Add To Cart
25mg 98% $270.00 Global Stock Buy Now Add To Cart
50mg 98% $458.00 Global Stock Buy Now Add To Cart
100mg 98% $825.00 Global Stock Buy Now Add To Cart
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Catalog Number AR01CC3P
Chemical Name 3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride,[2R-(2a,3a,4a,5b)]-
CAS Number 75172-81-5
Molecular Formula C6H14ClNO4
Molecular Weight 199.6327
MDL Number MFCD00269962
SMILES OC[C@H]1NC[C@@H]([C@H]([C@H]1O)O)O.Cl

Migalastat hydrochloride, also known as Galafold, is a pharmacological chaperone used in the treatment of Fabry disease. It functions by binding to mutant forms of the alpha-galactosidase A enzyme, which are responsible for the defects seen in Fabry disease patients. Through this mechanism, migalastat hydrochloride helps stabilize the enzyme, allowing it to traffic to the lysosomes where it can function more effectively in breaking down globotriaosylceramide (GL-3) and related substrates. This targeted approach is considered a significant advancement in the management of Fabry disease, offering a more specific and personalized treatment option for patients with certain genetic mutations. In chemical synthesis, migalastat hydrochloride can serve as a model compound for designing and developing other pharmacological chaperones. Its structure and mode of action provide valuable insights into the interactions between small molecules and target proteins, aiding in the rational design of new drug candidates for various genetic disorders. By studying the effects of migalastat hydrochloride on enzyme stability and functionality, researchers can better understand the principles of chaperone therapy and apply this knowledge to the development of novel treatments for other inherited metabolic diseases.
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