Genetics
<ѻýҕl class="page-description">News, opinions and meeting coverage in genetics.ѻýҕl>Latest in Genetics
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
U.S. clinics market oligonucleotide therapy for a wide range of conditions, without much evidence
Phase II study found brigatinib exceeded historical control by shrinking 10% of targeted tumors