Genetics
News, opinions and meeting coverage in genetics.
— 10% of patients in trials leading to eli-cel's approval developed MDS or AML
Latest in Genetics
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
FDA approves levacetylleucine (Aqneursa) for Niemann-Pick disease type C
Arimoclomol (Miplyffa) greenlit for ultra-rare genetic disorder
FDA approves levacetylleucine (Aqneursa) for Niemann-Pick disease type C
Arimoclomol (Miplyffa) greenlit for ultra-rare genetic disorder
More in Genetics
Findings "shift the dial more in favor of an animal origin," said one expert
Health news and commentary gathered by ֱ 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 ֱ staff
Poor air quality adds significantly to risk from genetic susceptibility factors
British data suggest how environmental and genetic factors add together
U.S. clinics market oligonucleotide therapy for a wide range of conditions, without much evidence
Moderate association between parental and offspring BMI at age 17
