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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….
3/ the family has to come up with the cash asap, before Mercan reaches 24 months, because timing is critical: "Infants dosed with #ZolgenSMA as soon as possible after diagnosis had better results than those who waited to be treated" (from curesma.org)
