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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

Two patients had rapid symptom improvement and reductions in malformation volume

Studies show 90% reduction in intravitreal treatment burden, durable effect on vision, anatomy

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