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Dr. Zeynep Hülya Gümüş 🌻 Profile picture
Dr. Zeynep Hülya Gümüş 🌻
@ZeynepHG
Associate Professor of Genetics and Genomics @IcahnInstitute & @SinaiImmunol & @TischCancer in NYC. Genetics, cancer, data sci & data viz @zeynep@genomic.social
Aug 13, 2020 • 19 tweets • 14 min read
1/ Hey Tweeps: meet Mercan! 7-month baby girl or a close friend. Mercan has spinal muscular atrophy (SMA1)- a muscle wasting disease that was considered incurable— now there is a cure! A one-time gene therapy by @Novartis that was recently @US_FDA approved! 2/ The drug is called #Zolgensma, and it is currently the world’s most expensive drug ever! $2.4 MILLION! npr.org/sections/healt….