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Amicus Therapeutics anticipates completing a rolling application by mid-year to seek approval of its investigational therapy AT-GAA for late-onset Pompe disease in the U.S. The announcement follows a pre-filing meeting with the U.S. Food and Drug Administration (FDA). The rolling biologics license application (BLA), initiated in…
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The U.S. Food and Drug Administration (FDA) has extended by three months its review of Sanofi Genzyme‘s application seeking approval of avalglucosidase alfa, its next-generation enzyme replacement therapy (ERT) for Pompe disease. The date for an agency decision regarding approval, previously set for May 18, is now Aug. 18. “Sanofi…
Fat replacement of back, abdominal, and trunk muscles in late-onset Pompe disease (LOPD) correlates with diaphragm weakness and worsening respiratory function, a long-term clinical study has determined. “These results suggest that identification of fat replacement in these areas … should…
While learning to care for her son, who has Pompe disease, columnist Keara Engle realizes her calling: a career in nursing.
Check out Pompe Disease News‘ collection of Health Insights, where you can find more information and guidance.
Pompe disease is a rare genetic disease characterized by the abnormal buildup of a sugar molecule called glycogen inside cells. Learn more about the disease here.
Pompe disease is an inherited condition caused by genetic mutations in the GAA gene, which provides instructions for the acid alpha-glucosidase enzyme. Learn more about what causes the disease here.
Muscle weakness is the predominant symptom of Pompe disease. Other symptoms depend on the type of Pompe patients have. Learn more about the symptoms of the disease here.
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