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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How a drugmaker converted the “abortion pill” into a lucrative orphan drug
The story of Korlym highlights how America’s drug development system can turn an old drug into a new one that treats relatively
few — but often very desperate — patients. -
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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FDA greenlights Sarepta Therapeutics’ milestone treatment for Duchenne muscular dystrophy
Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients.
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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.
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Payer’s Place: Dr. Anil Singh
Dr. Anil Singh shares his insights into the strategies employed by the organization to identify the most effective digital solutions for their members.
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Discover the Next-Gen Platform for Integrated Collaborative Care
Beyond EHRs and digital front doors, reducing the gaps in patient care journeys.
