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An Increased Emphasis on Individual Risk Factors and Goals
Zachary T. Bloomgarden, MD, MACE: We talked about lipid-lowering treatment and the notion that the idea of goals has recently been questioned. So, I certainly think that goals are a good way of treating cholesterol, but there are some controversies about that.
We use goals in treating all medical conditions. We use goals in treating blood pressure—we have to decide what the right blood pressure goal is for a given individual and then aim for that. We use goals in treating diabetes—we have to decide what the right A1C goal or fasting plasma glucose goal is for a given individual, and then stay with that. I’m completely in favor of the use of goals, as long as we do it in a rational fashion and apply it correctly.
STEER Data Open Door to SMA Gene Therapy for Wider Age Range of Children
March 19th 2025Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular atrophy (SMA) aged 2 to 17 years, who had previously been shut out of receiving gene therapy.
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Neurologists Share Tips for Securing Patient Access to Gene Therapies
March 19th 2025Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to make sure patients can access life-saving gene therapies for neuromuscular diseases.
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