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image: FDA Approves First Treatment for Spinal Muscular Atrophy

FDA Approves First Treatment for Spinal Muscular Atrophy

By | December 28, 2016

The oligonucleotide therapeutic will soon be available in the U.S.

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Oncologists have raised concerns about a mouse study that suggests the vaccine for human papillomavirus could cause brain damage.

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image: Gut Microbes Linked to Neurodegenerative Disease

Gut Microbes Linked to Neurodegenerative Disease

By | December 1, 2016

Bacteria in the intestine influence motor dysfunction and neuroinflammation in a mouse model of Parkinson’s disease.

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image: Oligonucleotide Therapeutics Near Approval

Oligonucleotide Therapeutics Near Approval

By | December 1, 2016

Successful late-stage clinical trials could mark the maturation of a new drug development platform, but the path to commercialization is not without hurdles.

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image: More Success Fixing Sickle Cell Gene with CRISPR

More Success Fixing Sickle Cell Gene with CRISPR

By | November 9, 2016

Researchers say they have sufficient in vitro and animal data to apply for human testing.

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image: New Support for Experimental Alzheimer’s Drug

New Support for Experimental Alzheimer’s Drug

By | November 2, 2016

Merck’s verubecestat, which is currently being tested in two Phase 3 studies, is safe, according to newly published Phase 1 data.

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Family members with a certain gene variant had unnecessary interventions, highlighting the potential hazards of precision medicine.

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image: Zika Update

Zika Update

By | October 24, 2016

Virus’s effect on RNA methylation; identifying brain cells targeted by Zika; virus found in vaginal secretions for two weeks after infection; updated CDC recommendations for Miami

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image: Nixing NETs to Prevent Metastasis

Nixing NETs to Prevent Metastasis

By | October 19, 2016

Researchers discover that neutrophil extracellular traps help cancers spread, and design enzyme-loaded nanoparticles to destroy them.

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Once implanted in mice, the edited stem cells produced normal hemoglobin.

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