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  • Therapeutic Options for the Management of Pompe Disease: Current . . .
    The year 2006 was game changing for the Pompe community as enzyme replacement therapy (ERT) with alglucosidase alfa (Myozyme Lumizyme ®, Sanofi-Genzyme) was approved for marketing by the Food and Drug Administration (FDA) and the European Medical Agency (EMA) 16 The first experiments with the aim to develop ERT for Pompe disease date as far
  • Pompe Disease: Symptoms Treatment - Cleveland Clinic
    Without treatment, infantile-onset Pompe disease is fatal early in childhood Many people with Pompe disease have breathing (respiratory) and heart problems Almost all people with the disease have muscle weakness Most people will need oxygen support and mobility assistance at some point in their lives
  • Pompe Disease - Symptoms, Causes, Treatment | NORD
    The treatment of Pompe disease is disease-specific, symptomatic and supportive Treatment requires the coordinated efforts of a team of specialists The input of pediatricians, internists, neurologists, orthopedists, cardiologists, dieticians, physical therapists and other healthcare professionals may be needed to develop the treatment plan
  • Pompe Disease | Glycogen Storage Disease Type II - Duke Health
    This lifesaving therapy was developed at Duke following clinical trials that allowed children with Pompe disease to access the therapy before it was widely available Enzyme replacement therapy has made it possible for people diagnosed with the disease to live longer, fuller lives We continue to actively research new and better ways to
  • New therapies for Pompe disease: are we closer to a cure?
    Disease-specific therapy is available for only a handful of neuromuscular disorders, including Pompe disease—a debilitating metabolic myopathy caused by a deficiency of the lysosomal glycogen-catabolising enzyme, acid α-glucosidase (GAA) Enzyme replacement therapy (ERT) with recombinant human GAA, alglucosidase alfa, was approved (by the US Food and Drug Administration and the European
  • Pompe Disease | Mount Sinai - New York - Mount Sinai Health System
    Pompe Disease Treatment While there is no cure for Pompe disease, treatment options are available Enzyme Replacement Therapy (ERT) is used to replace the enzyme acid alpha-glucosidase that is missing or not working properly in people with Pompe disease Lumizyme is the only FDA-approved treatment in the U S for the treatment of Pompe disease
  • Pompe Disease: Symtoms, Causes, Treatments - WebMD
    Nexviazyme, for those 1 year of age and older with late-onset Pompe disease; For adults with late-onset Pompe disease who have not been helped by enzyme-replacement therapy (ERT), the FDA has
  • About Pompe disease - Astellas Gene Therapies
    The only approved treatment for Pompe disease is enzyme replacement therapy, or ERT, which is a chronic treatment typically delivered in intravenous infusions every other week 1 ERT has markedly improved prognosis for patients with Pompe disease, 2 but patients may still develop significant symptoms due to limitations of ERT; patients may also





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