Targeting the Mutation, Not the Disease is Bringing Treatment to the Ultra-Rare Community
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Targeting the Mutation, Not the Disease is Bringing Treatment to the Ultra-Rare Community

The U.S. FDA just announced that it has accepted Genentech’s supplemental New Drug Application (sNDA) and has granted Priority Review for Zelboraf (vemurafenib) in patients with Erdheim-Chester disease from the…

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This Study Could Yield Urgently-Needed Data for the Prader-Willi Syndrome Community
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This Study Could Yield Urgently-Needed Data for the Prader-Willi Syndrome Community

Soleno Therapeutics, Inc. of Capnia reported in early July of this year that the company plans to start a Phase 3 clinical trial for evaluating diazoxide choline controlled-release (DCCR) in…

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Correcting Disease-Causing Genes in Humans: One Small Step for Humans, One Giant Leap for Humankind
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Correcting Disease-Causing Genes in Humans: One Small Step for Humans, One Giant Leap for Humankind

Genetic engineering has, for a long time, been a highly heated and contested issue. One must weigh the ethical ramifications and risks of engineering human embryos with the huge potential…

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