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Migalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder.It was developed by Amicus Therapeutics.The US Food and Drug Administration (FDA) granted it orphan drug status in 2004, [5] and the European Commission followed in 2006. [6]
Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, brain, and skin. [1] Fabry disease is one of a group of conditions known as lysosomal storage diseases .
Enzyme replacement therapy is available to treat mainly Fabry disease and Gaucher disease, and people with these types of sphingolipidoses may live well into adulthood. The other types are generally fatal by age 1 to 5 years for infantile forms, but progression may be mild for juvenile- or adult-onset forms.
Protalix BioTherapeutics , an Israeli company focused on the treatment of Gaucher disease and other rare diseases, recently reported its third-quarter earnings. But its core business -- a joint ...
4D Molecular Therapeutics Inc (NASDAQ: FDMT) has posted updated interim data from the Phase 1/2 trial of 4D-310 in Fabry disease at the 18th Annual WORLDSymposium. Following 4D-310 infusion, mean ...
Defects in the enzyme alpha-galactosidase lead to the buildup of globotriaosylceramide, causing Fabry's disease. [3] The pharmaceutical drug migalastat enhances the function of alpha-galactosidase and is used to treat Fabry's.
According to the New York Times, here's exactly how to play Strands: Find theme words to fill the board. Theme words stay highlighted in blue when found.
Robert J. Desnick is an American human geneticist whose basic and translational research accomplishments include significant discoveries in genomics, pharmacogenetics, gene therapy, personalized medicine, and the treatment of genetic diseases.