Genetics
News, opinions and meeting coverage in genetics.
— Benefits seen in patients with and without a genetic diagnosis
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
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 51˶ 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
Longer-acting crovalimab designed for more sustained effect and less treatment burden
Phase II study found brigatinib exceeded historical control by shrinking 10% of targeted tumors
Profitability, production cost, and low number of patients are barriers
Just one copy of the Christchurch variant conferred protection
Maternal history of memory impairment tied to higher amyloid burden
Researchers estimate the disorder may affect as many as one in 20,000 young people
Investigational treatment passes muster in phase III trial
