Article

Evaluating the Efficacy of Miglustat and Ambroxol Combination Therapy in a Phase 1b/2 Virtual Drug Trial for Type 1 Tay-Sachs Disease Using aiHumanoid Simulations

2024-11-22

Abstract excerpt

<h4>Abstract (Draft1)</h4> Type 1 Tay-Sachs Disease (TSD) is a rare and severe neurodegenerative disorder caused by HEXA Loss of Function (LOF) gene mutations, leading to the accumulation of lysosomal GM2 gangliosides and progressive neurological decline, with high lethality before age 10. Current treatments are primarily palliative, focusing on symptom management. This study investigates the therapeutic potential...

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Literature Corpus work: 8b9b29b4-da90-56a7-98c5-0005616fe79b
DOI: 10.1101/2024.11.21.24317709

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