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orphan drug
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Orphan Drug Development Is Next for Tech to Tackle
Drug developers need policies and technological infrastructure to execute on their goal of being patient-focused. At a minimum, this means a compliant way to engage patients and truly hear their voices; at its most effective, patient engagement also meets patients where they are – whenever and wherever that may be.
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FDA seals loophole that allowed some drugmakers to avoid pediatric clinical trials
PREA loophole had allowed companies to receive orphan drug designations for diseases common in adults.
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Payer’s Place: Dawn Maroney
Dawn Maroney, President, Markets of Alignment Health and CEO of Alignment Health Plan, to discuss how they are using technology to provide better service and care to consumers.
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Novartis adds gene therapy for spinal muscular atrophy with $8.7B acquisition of AveXis
The AveXis treatment for spinal muscular atrophy type 1 generated positive clinical data from a study in November last year — the 15 infants treated at 20 months with AVXS-10 suffered no adverse events.
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Alnylam stages a comeback with positive late-stage study results for RNAi treatment Patisiran
Promising Phase 3 study results for its RNAi treatment, called Patisiran, for a rare, genetic nerve disorder have increased the chances that Alnylam could be the first to commercialize a RNAi drug.
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Report: Orphan drugs cost 13.8 percent more per patient
The orphan drug industry is somewhat static in growth, but significantly outpaces the mass market drug industry, according to a new report from EvaluatePharma.
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Applying Remote Patient Monitoring to Surgery Prep and Recovery, Oncology and Women’s Health
Join us to learn about the latest trends in remote monitoring and how to extend its benefits beyond chronic conditions to more patients – all while using fewer staff resources.