Designation Based on Phase 2 SPRINT Trial in Pediatric Patients with NF1 Plexiform Neurofibromas Selumetinib Is a MEK 1/2 Inhibitor Being Co-Developed by AstraZeneca and Merck KENILWORTH, N.J.–(BUSINESS WIRE)– AstraZeneca and Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has granted…
AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US (known as MSD outside the US and Canada) today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1).
A team of scientists wants to accelerate research into a genetic disorder by using CRISPR to copy unique mutations from affected children into pigs.
When 3-year-old Gus Erickson was granted a wish by the Make-A-Wish organization, he told his mom and dad he wished for a piece of candy. Respecting this wish, his parents relayed it to Gus’ Make-A-Wish Minnesota coordinator. Her response: “Let’s give him some time to think of something bigger—and better—than…candy.” Gus was born with Neurofibromatosis…
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When Gregg and his wife, Becky, learned their son has Neurofibromatosis 6 years ago, they took a common first step for parents after receiving an NF1 diagnosis: they Googled “NF”. Online search results revealed imagery of patients with a relatively high burden of cutaneous neurofibromas (cNFs).
It is quickly becoming clear that “rocking out” to raise awareness and research funds for neurofibromatosis actually works. And it’s a whole lot of fun!
To help kids battling a rare disease, scientists forge a genetic link between people and pigs
Gus Erickson’s super power is making doors open with the wave of a finger.
It may sound silly, but the 5-year-old Rochester boy doesn’t yet understand the concept of automatic doors. His parents, Gregg and Becky, have fed his infatuation with super heroes by encouraging the harmless fun.
Life with an inherited disease sometimes brings unexpected twists and turns. Five-year-old Gus Erickson has navigated the gyrations with the help of Mayo Clinic’s Neurofibromatosis Clinic. Neurofibromatosis (NF) is a genetic disorder that causes tumors to form on nerve tissue.