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Disease of the Month – Fabry Disease

Shots: Fabry disease is a rare inherited neurological disorder that affects the body's ability to break down lipids into smaller components In this reprise of our Disease of the Month report, we bring an illuminating account of Fabry Disease with deep dive analysis of epidemiology, market size, disease management, available therapies, patient advocacy groups, and…

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VIEWPOINTS_Bettina Cockroft_2023

Bettina Cockroft, SVP & CMO at Sangamo Therapeutics Shares Insights from the Positive P-I/II Data for Fabry Disease Treatment

Shots:Bettina gave the details of the study design and shared the preliminary results from the P-I/II STAAR clinical study evaluating ST-920. The data were presented in an oral presentation at the 29th Congress of the European Society of Gene & Cell Therapy 2022 She also talked about enzyme replacement therapy (ERT) which is currently used for the…

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