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An RNAi Approval

The US Food and Drug Administration has approved Alnylam's Onpattro, an RNAi-based therapy, Stat News reports.

"This approval is part of a broader wave of advances that allow us to treat disease by actually targeting the root cause, enabling us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms," FDA Commissioner Scott Gottlieb says in a statement. "In this case, the effects of the disease cause a degeneration of the nerves, which can manifest in pain, weakness, and loss of mobility."

The agency approved Onpattro (patisiran) for the treatment of peripheral nerve disease in adults with hereditary transthyretin-mediated amyloidosis, a condition in which amyloid protein builds up in nerves, the heart, and other organs. This scope, Stat News notes, is narrower than what European regulators described, as it doesn't encompass the drug's effect on cardiac damage caused by the condition.

"Until today, there were no approved medications to treat these patients in the United States," Alnylam CEO John Maraganore said on a conference call, according to Stat News. "We do look forward to working with the FDA to expand the Onpattro label in the future."

Stat News adds the annual average list price of the drug will be $450,000.

The Scan

Wolf Howl Responses Offer Look at Vocal Behavior-Related Selection in Dogs

In dozens of domestic dogs listening to wolf vocalizations, researchers in Communication Biology see responses varying with age, sex, reproductive status, and a breed's evolutionary distance from wolves.

Facial Imaging-Based Genetic Diagnoses Appears to Get Boost With Three-Dimensional Approach

With data for more than 1,900 individuals affected by a range of genetic conditions, researchers compared facial phenotype-based diagnoses informed by 2D or 3D images in the European Journal of Human Genetics.

Survey Suggests Multigene Cancer Panel VUS Reporting May Vary Across Genetic Counselors

Investigators surveyed dozens of genetic counselors working in clinical or laboratory settings, uncovering attitudes around VUS reporting after multigene cancer panel testing in the Journal of Genetic Counseling.

Study Points to Tuberculosis Protection by Gaucher Disease Mutation

A mutation linked to Gaucher disease in the Ashkenazi Jewish population appears to boost Mycobacterium tuberculosis resistance in a zebrafish model of the lysosomal storage condition, a new PNAS study finds.