A major obstacle to enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) for Pompe disease is the development of high titers of anti-rhGAA antibodies in a subset of patients, which often leads to a loss of treatment efficacy. In an effort to induce sustained immune tolerance to rhGAA, we supplemented the rhGAA therapy with a weekly intravenous injection of synthetic vaccine particles carrying rapamycin (SVP-Rapa) during the first 3 weeks of a 12-week course of ERT in GAA-KO mice, and compared this with three intraperitoneal injections of methotrexate (MTX) per week for the first 3 weeks. Empty nanoparticles (NP) were used as negative control for SVP-Rapa. Co-administration of SVP-Rapa with rhGAA resulted in mor...
BackgroundLate-onset Pompe disease is a rare genetic neuromuscular disorder caused by lysosomal acid...
Pompe disease is a neuromuscular disease caused by an inherited deficiency of the lysosomal enzyme a...
Babies born with Pompe disease require life-long treatment with enzyme-replacement therapy (ERT). De...
Pompe disease results from acid α-glucosidase (GAA) deficiency, and enzyme replacement therapy (ERT)...
International audiencePompe disease results from acid α-glucosidase (GAA) deficiency and enzyme repl...
Pompe disease, which results from mutations in the gene encoding the glycogen-degrading lysosomal en...
<div><p>Enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) is the on...
Enzyme replacement therapy (ERT) is the current standard treatment for Pompe disease, a lysosomal st...
Enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) is the only FDA a...
International audienceEnzyme replacement therapy (ERT) is the standard-of-care treatment of Pompe di...
Enzyme and gene replacement strategies have developed into viable therapeutic approaches for the tre...
Cross-reactive immunological material-negative (CRIM-negative) infantile Pompe disease (IPD) patient...
Pompe disease is an autosomal recessive glycogen storage disorder caused by deficiency of the lysoso...
PURPOSE: To investigate immune tolerance induction with transient low-dose methotrexate (TLD-MTX) in...
Pompe disease is a neuromuscular disease caused by an inherited deficiency of the lysosomal enzyme a...
BackgroundLate-onset Pompe disease is a rare genetic neuromuscular disorder caused by lysosomal acid...
Pompe disease is a neuromuscular disease caused by an inherited deficiency of the lysosomal enzyme a...
Babies born with Pompe disease require life-long treatment with enzyme-replacement therapy (ERT). De...
Pompe disease results from acid α-glucosidase (GAA) deficiency, and enzyme replacement therapy (ERT)...
International audiencePompe disease results from acid α-glucosidase (GAA) deficiency and enzyme repl...
Pompe disease, which results from mutations in the gene encoding the glycogen-degrading lysosomal en...
<div><p>Enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) is the on...
Enzyme replacement therapy (ERT) is the current standard treatment for Pompe disease, a lysosomal st...
Enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) is the only FDA a...
International audienceEnzyme replacement therapy (ERT) is the standard-of-care treatment of Pompe di...
Enzyme and gene replacement strategies have developed into viable therapeutic approaches for the tre...
Cross-reactive immunological material-negative (CRIM-negative) infantile Pompe disease (IPD) patient...
Pompe disease is an autosomal recessive glycogen storage disorder caused by deficiency of the lysoso...
PURPOSE: To investigate immune tolerance induction with transient low-dose methotrexate (TLD-MTX) in...
Pompe disease is a neuromuscular disease caused by an inherited deficiency of the lysosomal enzyme a...
BackgroundLate-onset Pompe disease is a rare genetic neuromuscular disorder caused by lysosomal acid...
Pompe disease is a neuromuscular disease caused by an inherited deficiency of the lysosomal enzyme a...
Babies born with Pompe disease require life-long treatment with enzyme-replacement therapy (ERT). De...