Genetics
News, opinions and meeting coverage in genetics.
— Finding on HLA-DRB1*01:03 supports need for more and earlier genetic studies
Latest in Genetics
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
Higher risk for ever use, increasing duration of use but no increase in BRCA2 carriers
Agency appears unswayed by mitochondria-targeting drug for rare cardioskeletal disease
Higher risk for ever use, increasing duration of use but no increase in BRCA2 carriers
More in Genetics
FDA approves levacetylleucine (Aqneursa) for Niemann-Pick disease type C
Arimoclomol (Miplyffa) greenlit for ultra-rare genetic disorder
Findings "shift the dial more in favor of an animal origin," said one expert
Health news and commentary gathered by 鶹ý staff
Benefits seen in patients with and without a genetic diagnosis
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
Two patients had rapid symptom improvement and reductions in malformation volume
Studies show 90% reduction in intravitreal treatment burden, durable effect on vision, anatomy
Health news and commentary gathered by 鶹ý staff
Poor air quality adds significantly to risk from genetic susceptibility factors
British data suggest how environmental and genetic factors add together
