Treating a fatal muscle disease could be the next major advance for gene editing

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Nolan and Jack Willis and just 10 other boys took part in a clinical trial that led to the approval last fall of the very first drug to treat their rare, deadly muscle disease. Now the Willis boys are again test cases as a different type of medical question comes to the fore: whether insurers will cover the controversial drug, Exondys 51, which can cost more than $1 million a year even though it’s still unclear if it works

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In a confrontation between the hopes of desperate patients and clinical trial data, advisers to the Food and Drug Administration voted on Monday not to recommend approval of what would become the first drug for Duchenne muscular dystrophy

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“In an effort to influence the direction of DMD research, Parent Project Muscular Dystrophy…recently initiated and drafted guidance for the pharmaceutical industry,” Dr. Yoram Unguru, a Johns Hopkins bioethicist and pediatric hematologist/oncologist, noted in an article published in 2015

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Berman Institute Scholars launch a curriculum for training diverse healthcare workers to apply palliative care principles to pediatric patients suffering from sickle cell disease and Duchenne muscular dystrophy

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