Fabry disease (FD) is an X-linked lysosomal storage disorder resulting from the α-galactosidase A gene mutations. Enzyme-replacement-therapy (ERT) products for FD currently used include agalsidase alfa and agalsidase beta.
Fabry disease (FD) is an X-linked genetic disorder caused by the deficient activity of lysosomal α-galactosidase (α-Gal).
Background: Fabry disease is an X-linked lysosomal storage disorder caused by GLA mutations, resulting in α-galactosidase (α-Gal) deficiency and accumulation of lysosomal substrates.
Background: Fabry patients on reduced dose of agalsidase-beta or after switch to agalsidase-alfa show a decline in estimated glomerular filtration rate (eGFR) and an increase of the Mainz Severity Score Index.
Amicus Therapeutics and GSK announced the 6-month primary treatment period results from the first Phase III global registration study (Study...
Protalix BioTherapeutics announced positive 12-month on-treatment data from the first 16 out of the 22 adult patients with Fabry disease...
Protalix BioTherapeutics announced positive topline results from its Phase III BRIDGE clinical trial of PRX 102 (pegunigalsidase alfa), the Company's plant cell-expressed recombinant, PEGylated, cross-linked alpha-galactosidase-A product candidate under development for the treatment of Fabry disease.
BioTherapeutics and Chiesi announced final results from the BRIGHT Phase III clinical trial evaluating PRX 102 (pegunigalsidase alfa) for the potential treatment of Fabry disease.
The National Institute for Health and Care Excellence (NICE) in draft guidance acknowledged that Fabry disease is a serious condition...
Shire has withdrawn its Biologics License Application (BLA) for Replagal (agalsidase alfa) with the FDA. Shire has been in ongoing...