Top Tweets for #GENGLYCOS

14 days ago

FDA recently approved #GENGLYCOS by Ultragenyx - the FIRST #AAV #GeneTherapy to fix the root cause of glycogen storage disease type Ia (GSDIa), not just symptoms. A new era in genetic medicine begins.

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MedChemExpress

@MedChemExpress

about 1 month ago

🚨#FDA Approval Update | First approved therapy for Glycogen Storage Disease Type Ia The FDA has approved #Genglycos (pariglasgene brecaparvovec-opnr) for adults and children 8 years and older with #GSDIa, a rare inherited metabolic disorder. 🧬What’s behind GSDIa? A genetic mutation causes deficiency of glucose-6-phosphatase (#G6PC), an enzyme needed by the liver and kidneys to release glucose into the bloodstream during fasting. Without it, patients can experience dangerously low blood sugar when they go too long without eating. Until now, management has relied heavily on frequent meals and strict, around-the-clock cornstarch supplementation to prevent hypoglycemia. 💡What makes Genglycos different? This one-time AAV8-based gene therapy delivers a functional G6PC gene to the liver, aiming to restore the missing enzyme and help maintain blood glucose levels during fasting. 🚀A major milestone for gene therapy and rare metabolic diseases and a potential shift from managing symptoms to targeting the underlying cause. Read more: https://t.co/GxeqXOmaLc #GeneTherapy #RareDisease #GlycogenStorageDisease #MetabolicDisease #DrugDiscovery

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Top Tweets for #GENGLYCOS

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