Curated News: NEJM

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In this week's New England Journal of Medicine, researchers report results of a trial showing the efficacy of a new enzyme-replacement therapy for lysosomal acid lipase deficiency. In an accompanying editorial, Daniel J. Rader, MD, chair of the department of Genetics in the Perelman School of Medicine at the University of Pennsylvania, notes that this first-ever hepatocyte-targeting therapy will be pivotal in treating this disease.

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Imetelstat, a novel drug that targets telomerase, has demonstrated potential value in treating patients with myelofibrosis, according to the results of a study published today in the New England Journal of Medicine.

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A multinational team of physicians and scientists from City of Hope, the San Francisco Bay area and Europe recently reported success of a phase II clinical trial of a novel drug against essential thrombocythemia (ET), one of three myeloproliferative neoplasms (MPNs).

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In its first clinical trial, a breakthrough antibody therapy produced at least partial remissions in a third of patients with multiple myeloma who had exhausted multiple prior treatments, investigators at Dana-Farber Cancer Institute and other organizations report.

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Health care has much to learn from innovative high-tech companies, but not in the way most people think, according to a Perspective published today in the New England Journal of Medicine and authored by innovation experts from the Perelman School of Medicine and the Wharton School at the University of Pennsylvania. Innovation, they say, can most effectively achieve meaningful outcomes by testing many new ideas quickly, cheaply, and contextually.