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FDA advisors suggest the benefits of gene therapy outweigh risks of off-target editing

Test can "change the trajectory of these children's diseases"

Decision cites insufficient evidence of clinical meaningfulness

PSO should be performed early, particularly for those with the BRCA1 variant, researchers say

First-of-its-kind DNA test achieved 99% accuracy for variants in dozens of genes

Nedosiran approved for patients ages 9 years and older with primary hyperoxaluria type 1

FDA approves two-component therapy for patients not improving on enzyme replacement therapy

Outreach to families of minority patients may help identify genetic disorder

Clinical meaningfulness is a different question, however

Lung function, BMI, bacterial swabs, sweat chloride levels also improved

Pilot study suggests early testing for seizures, regardless of treatment setting

Pozelimab reduced hospitalizations, albumin transfusions in CHAPLE disease patients

Early-phase data suggest a dose that can keep bilirubin in check without hours of phototherapy

Shift away from blanket restrictions is not placing top-tier athletes at undue risk, data suggest