Genetics
News, opinions and meeting coverage in genetics.
Latest in Genetics
Finding on HLA-DRB1*01:03 supports need for more and earlier genetic studies
Panelists acknowledge difficulty generating stronger data in ultra-rare disease
10% of patients in trials leading to eli-cel's approval developed MDS or AML
Agency appears unswayed by mitochondria-targeting drug for rare cardioskeletal disease
10% of patients in trials leading to eli-cel's approval developed MDS or AML
Agency appears unswayed by mitochondria-targeting drug for rare cardioskeletal disease
More in Genetics
Phase III trial falls short of efficacy target, but positive signal emerges in younger kids
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
Studies show 90% reduction in intravitreal treatment burden, durable effect on vision, anatomy