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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.
Pegunigalsidase alfa, sold under the brand name Elfabrio, is an enzyme replacement therapy for the treatment of Fabry disease. [2] [4] It is a recombinant human α-galactosidase-A. [4] It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme. [2] The most common side effects are infusion-related reactions, hypersensitivity and ...
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 ...
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 ...
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 is a rare inherited disease linked to the X chromosome and is estimated to affect roughly 1 in 117,000 people in the general population. About Genzyme, a Sanofi Company
In this treatment, the accumulated compounds are inhibited from forming in the body of a patient with a lysosomal storage disease. [10] The accumulated compounds are responsible for the symptoms of these disorders, and they form via a multi-step biological pathway. [ 10 ]
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