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Efficacy and safety of enzyme-replacement-therapy with agalsidase alfa in 36 treatment-naive Fabry disease patients.
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.
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.
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.
Fabry disease under enzyme replacement therapy-new insights in efficacy of different dosages.
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.
Phase III MODIFY study of lucerastat fails to meet primary endpoint in Fabry disease.
Idorsia announced that MODIFY, the Phase III study to investigate the effect of lucerastat, as an oral substrate reduction therapy for the treatment of adult patients with Fabry disease, did not meet the primary endpoint.
Amicus Therapeutics announces European Commission approval of Galafold for adolescents with Fabry Disease.
Amicus Therapeutics has announced the European Commission has approved Galafold (migalastat) for use in adolescents aged 12 to <16 years weighing greater than 45 kg with a confirmed diagnosis of fabry disease and who have an amenable mutation.