DCCR Tablets for GSD1A Granted Orphan Drug Status
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DCCR Tablets for GSD1A Granted Orphan Drug Status

In a news release from June 2, 2021, biopharmaceutical company Soleno Therapeutics, Inc. ("Soleno") shared an exciting update. The company's investigational treatment, DCCR (diazoxide choline) Extended-Release tablets, received Orphan Drug…

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New Data Available for GSD1a, OTC Deficiency, and Wilson Disease Studies
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New Data Available for GSD1a, OTC Deficiency, and Wilson Disease Studies

  In a recent press release, biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx") shared positive safety and efficacy data from Phase 1/2 clinical trials on gene therapy solutions for ornithine transcarbamylase…

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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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The First Patient Has Been Dosed on a Clinical Trial of an Investigational Gene Therapy for the Treatment of GSDIa
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The First Patient Has Been Dosed on a Clinical Trial of an Investigational Gene Therapy for the Treatment of GSDIa

According to a source article that can be found here on Ultragenyx’s website, the first patient has been dosed in a trial of an investigational gene therapy called DTX401 that…

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