Efficient Process development strategies for prevalent “rare disease” drugs
Temp 6
DST
July 5, 2023
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Rare disorder
Invito study
Quantitative data
• Process development for the cysticfibrosis drug Tezacaftor.
• Process development for thelysosomal disorder drug Eliglustat
(NIPER-Kolkata)
• Process development of existing ASO-based Duchenne Muscular Dystrophy drug Eteplirsen to make it affordable (IACS, Kolkata; and NIPER-Kolkata).
Cystic fibrosis, Tezacaftor, Eliglustat, Lysosomal disorder, Eteplirsen, Duchenne Muscular Dystrophy, Rare disorder
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Prof. Ravichandiran Velayutham/None/NIPER, Kolkata