Small molecule drug discovery for rare genetic disorder therapy
Temp 3
DST
Nov. 2, 2023
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Rare genetic disorder
In vitro study
Quantitative data
• Lead molecule identification, development, and validation for a rare neuromuscular genetic disorder, GNE Myopathy. The study can be extrapolated for other rare genetic disorders.
• Identification of novel chemical small effector compounds for the treatment of GNE Myopathy that may further be explored for clinical trial study in India
Drug Discovery, Rare genetic disorders, GNE Myopathy, Lead molecule identification
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Dr. Ranjana Arya/None/JNU, Delhi