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gene therapy

Priciest Yet

Novartis's newly approved Zolgensma, a gene therapy for spinal muscular atrophy, is to cost $2.1 million, the AP says.

Looking Into It

A second death in gene therapy trial for type 1 spinal muscular atrophy is under investigation, according to Reuters.

St. Jude Children's Research Hospital scientists have treated infants with X-linked severe combined immunodeficiency using gene therapy in an early phase study.

The New York Times reports on new gene therapy efforts to treat sickle-cell disease.

Positive Review

A Canadian panel recommends public coverage of the gene therapy Kymriah if its cost comes down, the Globe and Mail reports.

New Scientist reports that researchers have tested an ALS gene therapy in a mouse model of disease.

The San Diego Union-Tribune reports on the difficulties health insurers face with new gene and cell therapies.

The researchers characterized how gene therapy affects the immune cell repertoires and microbiomes of X-linked severe combined immunodeficiency patients.

The Wall Street Journal looks into the cost of new gene therapies.

This Week in PNAS

In PNAS this week: DNA vaccine-based approach for Duchenne muscular dystrophy tested in mice, antimicrobial found in bear saliva, and more.

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US National Institutes of Health Director Francis Collins says he will avoid male-only speaker panels.

Two patients fell ill, and one subsequently died, following a fecal microbiome transplant that harbored multi-drug-resistant bacteria, according to the New York Times.

Technology Review reports that eGenesis is testing whether organs from genetically modified pigs can be transplanted into monkeys.

In Science this week: almond reference genome, and more.