A UConn Health Team Administers the World’s First Gene Therapy for Glycogen Storage Disease Type-1a

In January of this year, a University of Connecticut (UConn) team administered the world’s first infusion of a new gene therapy as part of a long-awaited clinical trial. According to…

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Experimental Treatment for Glycogen Storage Disease Type III Gets Orphan Designation in EU and US
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Experimental Treatment for Glycogen Storage Disease Type III Gets Orphan Designation in EU and US

According to a story from Market Watch, the biopharmaceutical company Ultragenyx Pharmaceutical, Inc. recently announced that it was given Orphan Drug designation from both the US Food and Drug Administration…

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Life as a Parent of a Child With Glycogen Storage Disease: A Rare Disease Story From the Czech Republic
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Life as a Parent of a Child With Glycogen Storage Disease: A Rare Disease Story From the Czech Republic

When your child is constantly at risk of hypoglycemia and their health and life is threatened in the case of any major problem, you have no other choice but to…

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