Genetics
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Phase III trial falls short of efficacy target, but positive signal emerges in younger kids
If approved, this would be the first drug for Niemann-Pick disease type C
This is the second time the drug has been submitted to the FDA for Niemann-Pick disease type C
In randomized study, guided site-specific therapy improved PFS versus empirical chemotherapy
This is the second time the drug has been submitted to the FDA for Niemann-Pick disease type C
In randomized study, guided site-specific therapy improved PFS versus empirical chemotherapy
More in Genetics
Studies show 90% reduction in intravitreal treatment burden, durable effect on vision, anatomy
![ASRS over a photo of Stockholmsmässan Convention Center in Stockholm, Sweden.](http://assets.medpagetoday.net/media/images/111xxx/111137.jpg)
U.S. clinics market oligonucleotide therapy for a wide range of conditions, without much evidence
![A computer rendering of an oligonucleotide aptamer attacking a cancer cell.](http://assets.medpagetoday.net/media/images/110xxx/110966.jpg)
Phase II study found brigatinib exceeded historical control by shrinking 10% of targeted tumors
![A computer rendering of a meningioma.](http://assets.medpagetoday.net/media/images/110xxx/110772.jpg)