Unravelling the mechanism of action of enzyme replacement therapy in Fabry disease.
Seoul, South Korea. In J Hum Genet, Nov 2015
UNASSIGNED: Fabry disease (FD) is a rare X-linked recessive glycosphingolipid-storage disorder caused by deficient activity of the lysosomal enzyme alpha-galactosidase A. Intravenous enzyme replacement therapy (ERT) has been used to supplement deficient enzyme activity in patients with FD.
Glatiramer Acetate 40 mg/mL in Relapsing-Remitting Multiple Sclerosis: A Review.
Auckland, New Zealand. In Cns Drugs, May 2015
In the randomized, phase III GALA study in patients with relapsing-remitting MS (RRMS), glatiramer acetate 40 mg/mL three times weekly reduced annualized relapse rates significantly more than placebo, and indirect comparisons indicate that the efficacy of the three-times-weekly regimen is similar to that of the 20 mg/mL once-daily regimen.
Fabry disease and the heart.
Würzburg, Germany. In Best Pract Res Clin Endocrinol Metab, Mar 2015
Fabry disease is induced by a mutation in the alpha-galactosidase A gene, causing a deficiency of the enzyme alpha-galactosidase A. (1) The enzyme defect leads to progressive intracellular accumulation of globotriaosylceramide in lysosomes of various tissues and organs, including heart, kidney and nerve system.
Enzymes approved for human therapy: indications, mechanisms and adverse effects.
Australia. In Biodrugs, Feb 2015
The introduction and regulatory approval of 20 different recombinant enzymes has enabled, often for the first time, effective enzyme-replacement therapy for some lysosomal storage disorders, including Gaucher (imiglucerase, taliglucerase, and velaglucerase), Fabry (agalsidase alfa and beta), and Pompe (alglucosidase alfa) diseases and mucopolysaccharidoses I (laronidase), II (idursulfase), IVA (elosulfase), and VI (galsulfase).
Cincinnati, United States. In Lancet, 2008
Fabry's disease is an X-linked lysosomal storage disorder caused by abnormalities in the GLA gene, which leads to a deficiency in alpha-galactosidase A. The consequent abnormal accumulation of glycosphingolipids results in several clinical signs and symptoms and substantial morbidity and mortality.