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Top of the morning to you. Another busy day is unfolding here on the Pharmalot campus, where we are mulling over our cluttered to-do list and plotting our next moves. No doubt, you can relate. After all, this is only the middle of the week and there is so much to be done. This calls, as you may imagine, for a hot cup of stimulation. We are switching to glazed doughnut today — you can never be too sweet, yes? Feel free to join us. Meanwhile, here are some tidbits. The world is a busy place, yes? Hope you make your way today. …

The Food and Drug Administration rejected a one-time gene therapy for hemophilia A, delaying what would have been a major medical milestone in a decision that upended the expectations of doctors, patients, and Wall Street, STAT reports. The FDA move means the gene therapy from BioMarin Pharmaceutical (BMRN), which promised to revolutionize the treatment of the bleeding disorder, will be delayed until at least 2022. The FDA is unconvinced BioMarin demonstrated the therapy’s durability and wants two more years of data from an ongoing study.

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