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Gene therapy for long-term treatment of Pompe disease

Gene therapy for long-term treatment of Pompe disease

Unmet Need Pompe disease is a rare heritable disorder impacting heart and skeletal muscle function with an estimated incidence rate of 1 in 40,000 births in the US. Of these affected births, a third take…

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A method of screening and monitoring glycogen storage diseases with a single biomarker

A method of screening and monitoring glycogen storage diseases with a single biomarker

Unmet Need Glycogen storage disease type I (GSD I) is an autosomal recessive inherited disorder of carbohydrate metabolism associated with life threatening fasting hypoglycemia, and significant morbidity and mortality. Currently, the standard of care therapy…

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