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Dr Aimee Tharaldson on the Interest in Developing Orphan Drugs
Orphan medications are gaining a lot of focus from manufacturers, Aimee Tharaldson, PharmD, senior clinical consultant for emerging therapeutics at Express Scripts, said at the Academy of Managed Care Pharmacy's 27th Annual Meeting & Expo in San Diego, April 7-10.
Orphan medications are gaining a lot of focus from manufacturers, Aimee Tharaldson, PharmD, senior clinical consultant for emerging therapeutics at Express Scripts, said at the Academy of Managed Care Pharmacy's 27th Annual Meeting & Expo in San Diego, April 7-10.
These drugs treat populations of less than 200,000 patients, but manufacturers are given financial incentives to develop orphan medications and as a result in 2014, 75% of specialt drug approvals were for orphan drugs.
"These medications cost hundreds of thousands of dollars a year and oftetimes about 30% of orphan medications become blockbuster medications," Dr Tharaldson said.
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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The Importance of Examining and Preventing Atrial Fibrillation
August 29th 2023At this year’s American Society for Preventive Cardiology Congress on CVD Prevention, Emelia J. Benjamin, MD, ScM, delivered the Honorary Fellow Award Lecture, “The Imperative to Focus on the Prevention of Atrial Fibrillation,” as the recipient of this year’s Honorary Fellow of the American Society for Preventive Cardiology award.
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Promoting Equity in Public Health: Policy, Investment, and Community Engagement Solutions
June 28th 2022On this episode of Managed Care Cast, we speak with Georges C. Benjamin, MD, executive director of the American Public Health Association, on the core takeaways of his keynote session at AHIP 2022 on public health policy and other solutions to promote equitable health and well-being.
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How Access to SMA Treatment Varies Globally and by Insurance Type
March 18th 2025Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic advances in treating spinal muscular atrophy (SMA) are not uniformly making it into the hands of patients who could benefit.
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