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First RNAi Drug Hits the Market — At a Very High Price

On August 13, the U.S. Food and Drug Administration (FDA) approved Alnylam’s Onpattro (patisiran), rapidly followed by that product’s approval in Europe three weeks later.  Onpattro, which we previously wrote about, is the first RNAi-based treatment to reach the marketplace. It is intended to treat peripheral nerve disease caused by a rare genetic condition called...

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Innovations in Electronic Health Records

Increasingly, hospital and health systems have moved from paper charts to electronic health records (EHRs). EHRs offer ways to streamline clinical workflows, reduce medical errors and increase patient safety, and provide stronger support for clinical decision-making. However, linking such records from different locations for the same patient has proved problematic in many cases, leading to...

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Gene Therapy Update Part 2

Last week we wrote about recent news in the field of CRISPR/Cas9 research. But CRISPR is not the only approach to gene editing under development, nor the most advanced.   First Human Gene Editing Data — But Not from CRISPR   Sangamo Therapeutics, Inc. has been developing an approach to gene editing based on zinc...

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Gene Therapy Update Part 1: Big Advances and Big Questions for CRISPR

The gene therapy field has seen several developments in recent months, especially around CRISPR, with both significant achievements and further controversies about the safety of the gene editing approach.   CRISPR Cure for Duchenne’s Muscular Dystrophy (DMD) in Dogs   Eric Olson’s team of researchers at the University of Texas Southwestern’s Harmon Center for Regenerative...

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Seminal Immuno-oncology Discoveries Honored by 2018 Nobel Prize

Seemingly spontaneous remissions from terminal cancers are known to occur on rare occasions.  For over 100 years, this observation has spurred scientists to attempt to engage the immune system to fight cancer, with little success. But on October 1, the 2018 Nobel Prize in physiology and medicine was awarded to two scientists whose seminal discoveries...

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The Growing Influence of ICER

We have previously written about the Institute for Clinical and Economic Review (ICER), the independent watchdog whose mission is to offer an objective evaluation of the clinical and economic value of prescription drugs, medical tests, and other healthcare innovations.   Many European nations have governmental or quasi-governmental agencies, like the United Kingdom’s NICE (the National...