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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 .
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 ...
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.
The company prepares to initiate the expected enrollment of patients in the Phase 1/2 study of ST-503 for idiopathic small fiber neuropathy in mid-2025 and file an anticipated Clinical Trial ...
The bacterial Shiga toxin can be used for targeted therapy of certain gastrointestinal cancers that express the receptor of the Shiga toxin. [4] For this purpose a non-specific chemotherapeutic agent is conjugated to the B-subunit to make it specific. In this way only the tumor cells, but not healthy cells, should be destroyed during therapy. [5]
The therapy, branded as Ryoncil, is the first mesenchymal stromal cell therapy approved to treat pediatric patients aged two months and older whose GVHD symptoms have not responded to standard ...
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