GlaxoSmithKline says it has treated a child with Strimvelis, its gene therapy for immune deficiency

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Even as high drug prices make headlines, the challenge of getting sick children the kind of medication they can take and tolerate — often by creating liquid formulations of drugs that are already on the market — is seen by some companies as a lucrative opportunity

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This was the proposal: Deliberately infect a small group of consenting adults with the Zika virus to learn about the disease and speed up the search for a vaccine. The need is clear. Zika is an emerging global threat to public health. The disease can be devastating, especially for the babies of mothers who catch it while pregnant

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Spark Therapeutics is within striking distance of a landmark green light from the FDA for its treatment for certain forms of blindness

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Drugmaker Sarepta Therapeutics won a big victory when its $300,000 muscular dystrophy drug was recently approved, but the company had other reasons to celebrate, too. They were also awarded the drug world’s equivalent of a Willy Wonka golden ticket

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More than 21,000 complaints have been lodged against his Wen Hair Care, and Mr. Dean, the blue-eyed, golden-haired stylist to the stars, has found himself at the center of a fierce debate over the government’s power to ensure the safety of a cosmetics industry with about $50 billion in annual sales

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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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