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Glycogen Storage Disease Research

David Weinstein, MD MSc, is the world’s top researcher for Glycogen Storage Disease (GSD). In July 2018, he launched the world’s first human gene therapy clinical trial to treat GSD – an advance 20 years in the making. Currently, there is no cure for GSD and no approved pharmacological therapies.

Early Results of the Gene Therapy Clinical Trial to Treat GSD

  • Positive topline safety and efficacy data from the first, lowest dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV) based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa).
  • A biologic response, reflected by improved glucose control and increased time to hypoglycemia during fasting, was observed in all three patients, with two patients demonstrating a clinically meaningful improvement in time to hypoglycemia during a controlled fasting challenge.
  • In addition to the promising results observed with the controlled fasting challenge, all three patients demonstrated improvement in glucose control throughout the day, and all three patients have been able to decrease their daily cornstarch intake by approximately half.
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